Decision
Orphan register
Updated 3 November 2025
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| Product name | Active substance | Orphan Condition | OD Indication | Designation number | Orphan Market Exclusivity Expiry date |
|---|---|---|---|---|---|
| ABECMA | idecabtagene vicleucel | Multiple myeloma | Abecma is indicated for the treatment of adult patients with relapsed and refractory multiple myeloma who have received at least three prior therapies, including an immunomodulatory agent, a proteasome inhibitor and an anti-CD38 antibody and have demonstrated disease progression on the last therapy | PLGB 50412/0023/OD1 | 23-Jun-32 |
| Adzynma | recombinant human A disintegrin and metalloproteinase with thrombospondin motifs 13 (rADAMTS13) | Thrombotic thrombocytopenic purpura (TTP) | Adzynma as an enzyme replacement therapy indicated for the treatment of ADAMTS13 deficiency in children and adult patients with congenital thrombotic thrombocytopenic purpura of all age groups | PL 16189/0148-0149/OD1 | 11-May-35 |
| AGAMREE | VAMOROLONE | Duchenne muscular dystrophy | Vamorolone in the treatment of Duchenne muscular dystrophy (DMD) in patients aged 4 years and older. | PLGB 25835/0005/OD1 | 11-Jan-34 |
| Akantior | POLIHEXANIDE | Acanthamoeba keratitis | Akantior is indicated for the treatment of acanthamoeba keratitis in adults and children from 12 years of age | PL 36078/0007/OD1 | 15-May-35 |
| ALPROLIX | eftrenonacog alfa Recombinant fusion protein consisting of human coagulation factor IX attached to the Fc domain of human IgG1 | Haemophilia B (congenital factor IX deficiency) | Treatment and prophylaxis of bleeding in patients with haemophilia B (congenital factor IX deficiency). ALPROLIX can be used for all age groups. | PLGB 30941/0003 – 0007/OD1 | 13-May-26 |
| ALYFTREK | TEZACAFTOR, DEUTIVACAFTOR, VANZACAFTOR CALCIUM DIHYDRATE | Orphan condition: cystic fibrosis (CF) | Alyftrek 50mg/20mg/4mg & 125mg/50mg/10mg film-coated tablets for the treatment of cystic fibrosis (CF) in people aged 6 years and older who have at least one F508del mutation or another responsive mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. | PL 22352/0024-25/OD1 | 07-Mar-35 |
| AMGLIDIA | Glibenclamide | Neonatal diabetes | AMGLIDIA is indicated for the treatment of neonatal diabetes mellitus, for use in newborns, infants and children. Sulphonylureas like AMGLIDIA have been shown to be effective in patients with mutations in the genes coding for the ?-cell ATP-sensitive potassium channel and chromosome 6q24-related transient neonatal diabetes mellitus. | PLGB 50687/0001 – 0004/OD1 | 28-May-28 |
| ARIKAYCE liposomal | Amikacin sulfate | Non-tuberculous mycobacterial lung disease | ARIKAYCE liposomal is indicated for the treatment of non-tuberculous mycobacterial (NTM) lung infections caused by Mycobacterium avium Complex (MAC) in adults with limited treatment options who do not have cystic fibrosis. | PLGB 47434/0001/OD1 | 28-Oct-30 |
| Artesunate Amivas | Artesunate | Malaria | Artesunate Amivas is indicated for the initial treatment of severe malaria in adults and children | PLGB 55184/0002 /OD1 | 27-Mar-32 |
| ASPAVELI | pegcetacoplan | Paroxysmal nocturnal haemoglobinuria | ASPAVELI is indicated in the treatment of adult patients with paroxysmal nocturnal haemoglobinuria (PNH) who are anaemic after treatment with a C5 inhibitor for at least 3 months. | PLGB 30941/0022/OD1 | 24-Feb-32 |
| Aucatzyl | autologous T cells expressing an anti-CD19 chimeric antigen receptor. Autologous T cells that are genetically modified ex-vivo with a lentiviral vector to express a novel CD19 chimeric antigen receptor. | Acute lymphoblastic leukaemia | Aucatzyl is indicated for the treatment of adult patients (?18 years) with relapsed or refractory B cell precursor acute lymphoblastic leukaemia | PLGB 46113/0001/OD1 | 25-Apr-35 |
| Avacopan Vifor | avacopan | Granulomatosis with polyangiitis | Avacopan Vifor, in combination with a rituximab or cyclophosphamide regimen, is indicated for the treatment of adult patients with severe, active granulomatosis with polyangiitis (GPA) or microscopic polyangiitis (MPA) | PLGB 50784/0008/OD1 | 05-May-32 |
| Avacopan Vifor | avacopan | Microscopic polyangiitis | Avacopan Vifor, in combination with a rituximab or cyclophosphamide regimen, is indicated for the treatment of adult patients with severe, active granulomatosis with polyangiitis (GPA) or microscopic polyangiitis (MPA) | PLGB 50784/0008/OD2 | 05-May-32 |
| AYVAKYT | avapritinib (S)-1-(4-fluorophenyl)-1-(2-(4-(6-(1-methyl-1H-pyrazol-4-yl)pyrrolo[2,1-f][1,2,4]triazin-4-yl)piperazin-yl)pyrimidin-5-yl)ethan-1-amine | Gastrointestinal stromal tumours | AYVAKYT is indicated as monotherapy for the treatment of adult patients with unresectable or metastatic gastrointestinal stromal tumours (GIST) harbouring the platelet-derived growth factor receptor alpha (PDGFRA) D842V mutation | PLGB 52115/0001 – 0003/0D1 | 25-Sep-30 |
| AYVAKYT | avapritinib (S)-1-(4-fluorophenyl)-1-(2-(4-(6-(1-methyl-1H-pyrazol-4-yl)pyrrolo[2,1-f][1,2,4]triazin-4-yl)piperazin-yl)pyrimidin-5-yl)ethan-1-amine | Mastocytosis | AYVAKYT is indicated as monotherapy for the treatment of adult patients with aggressive systemic mastocytosis (ASM), systemic mastocytosis with an associated haematological neoplasm (SM-AHN), or mast cell leukaemia (MCL). | PLGB 52115/0001 – 0002/OD2, PLGB 52115/0006 – 0007/OD2 | 10-Sep-34 |
| Besponsa | Inotuzumab ozogamicin | B-cell acute lymphoblastic leukaemia | BESPONSA is indicated as monotherapy for the treatment of adults with relapsed or refractory CD22-positive B cell precursor acute lymphoblastic leukaemia (ALL). Adult patients with Philadelphia chromosome positive (Ph+) relapsed or refractory B cell precursor ALL should have failed treatment with at least 1 tyrosine kinase inhibitor (TKI). | PLGB 00057/1546/OD1 | 03-Jul-27 |
| BLINCYTO | Blinatumomab | Acute lymphoblastic leukaemia | BLINCYTO is indicated as monotherapy for the treatment of adults with CD19 positive relapsed or refractory B-cell precursor acute lymphoblastic leukaemia (ALL). Patients with Philadelphia chromosome positive B-cell precursor ALL should have failed treatment with at least 2 tyrosine kinase inhibitors (TKIs) and have no alternative treatment options. BLINCYTO is indicated as monotherapy for the treatment of adults with Philadelphia chromosome negative CD19 positive B-cell precursor ALL in first or second complete remission with minimal residual disease (MRD) greater than or equal to 0.1%. BLINCYTO is indicated as monotherapy for the treatment of paediatric patients aged 1 year or older with Philadelphia chromosome negative CD19 positive B-cell precursor ALL which is refractory or in relapse after receiving at least two prior therapies or in relapse after receiving prior allogeneic haematopoietic stem cell transplantation. BLINCYTO is indicated for the treatment of adult patients with Philadelphia chromosome negative CD19-positive B-cell precursor leukaemia ALL in the consolidation phase. BLINCYTO is indicated for the treatment of paediatric patients aged 1 year or older with Philadelphia chromosome negative CD19-positive B-cell precursor leukaemia ALL in first relapse in the consolidation phase. | PLGB 13832/0018/OD1 | 25-Nov-27 |
| Brineura | cerliponase alfa Recombinant human tripeptidyl-peptidase 1 | Neuronal ceroid lipofuscinosis type 2 disease | Brineura is indicated for the treatment of neuronal ceroid lipofuscinosis type 2 (CLN2) disease, also known as tripeptidyl peptidase 1 (TPP1) deficiency. | PLGB 45814/0001/OD1 | 01-Jun-27 |
| Bylvay | Not set | Progressive familial intrahepatic cholestasis | Bylvay is indicated for the treatment of progressive familial intrahepatic cholestasis (PFIC) in patients aged 6 months or older. | PLGB 36216/0001-0004/OD1 | 07-Sep-31 |
| Cablivi | caplacizumab Nanobody directed towards the human A1 domain of von Willebrand factor | Thrombotic thrombocytopenic purpura | Cablivi is indicated for the treatment of adults and adolescents of 12 years of age and older weighing at least 40 kg experiencing an episode of acquired thrombotic thrombocytopenic purpura (aTTP), in conjunction with plasma exchange and immunosuppression. | PLGB 04425/0888/OD1 | 04-Sep-30 |
| Carvykti | Ciltacabtagene autoleucel | Multiple myeloma | Treatment of adult patients with relapsed and refractory multiple myeloma, who have received at least one prior therapy, including an immunomodulatory agent and a proteasome inhibitor, have demonstrated disease progression on the last therapy, and are refractory to lenalidomide. | PLGB 00242/0745/OD1 | 04-Jan-33 |
| Casgevy | autologous CD34+ hematopoietic stem cells with a CRISPR-edited erythroid enhancer region of the BCL11A gene | ? thalassaemia intermedia and major | Authorised orphan indication: treatment of transfusion-dependent ?-thalassemia in patients 12 years of age and older for whom a human leukocyte antigen-matched related haematopoietic stem cell donor is appropriate and a human leukocyte antigen matched related haematopoietic stem cell donor is not available. | PLGB 22352/0019/OD1 | 15-Nov-33 |
| Casgevy | autologous CD34+ hematopoietic stem cells with a CRISPR-edited erythroid enhancer region of the BCL11A gene | sickle cell disease | treatment of sickle cell disease in patients 12 years of age and older with recurrent vaso-occlusive crises who have the ?S/?S, ?S/?+ or ?S/?0 genotype, for whom a haematopoeitic stem cell transplantation is appropriate and a human leukocyte antigen matched related haematopoietic stem cell donor is not available. | PLGB 22352/0019/OD2 | 15-Nov-33 |
| Cerdelga | eliglustat | Gaucher Disease | Cerdelga is indicated for the long-term treatment of adult patients with Gaucher disease type 1 (GD1), who are CYP2D6 poor metabolisers (PMs), intermediate metabolisers (IMs) or extensive metabolisers (EMs). | PLGB 04425/0763/OD1 | 21-Jan-27 |
| Chenodeoxycholic acid Leadiant | Chenodeoxycholic acid | Inborn errors of primary bile acid synthesis | Chenodeoxycholic acid is indicated for the treatment of inborn errors of primary bile acid synthesis due to sterol 27 hydroxylase deficiency (presenting as cerebrotendinous xanthomatosis (CTX)) in infants, children and adolescents aged 1 month to 18 years and adults | PLGB 44975/0001/OD1 | 12-Apr-27 |
| Coagadex | Human coagulation factor X | Hereditary factor X deficiency | Coagadex is indicated for the treatment and prophylaxis of bleeding episodes and for perioperative management in patients with hereditary factor X deficiency. | PLGB 08801/0059 – 0060/OD1 | 18-Mar-28 |
| Cresemba | isavuconazole | Mucormycosis | Treatment of mucormycosis in patients from 1 year of age for whom amphotericin B is inappropriate. | PLGB 32205/0005 – 0006/OD1 | 19-Oct-27 |
| Cresemba | isavuconazole | Invasive aspergillosis | Treatment of invasive aspergillosis in patients from 1 year of age. | PLGB 32205/0005 – 0006/OD2 | 19-Oct-27 |
| CRYSVITA | Burosumab Recombinant human monoclonal IgG1 antibody for fibroblast growth factor 23 | X-linked hypophosphataemia | CRYSVITA is indicated for the treatment of X-linked hypophosphataemia with radiographic evidence of bone disease in children 1 year of age and older and adolescents with growing skeletons | PLGB 50262/0001/OD1 | 21-Feb-28 |
| Cystadrops | mercaptamine Cysteamine hydrochloride | Cystinosis | Cystadrops is indicated for the treatment of corneal cystine crystal deposits in adults and children from 2 years of age with cystinosis. | PLGB 15266/0021/OD1 | 23-Jan-27 |
| Darzalex | Daratumumab | Plasma cell myeloma | • in combination with lenalidomide and dexamethasone or with bortezomib, melphalan and prednisone for the treatment of adult patients with newly diagnosed multiple myeloma who are ineligible for autologous stem cell transplant • in combination with bortezomib, lenalidomide and dexamethasone for the treatment of adult patients with newly diagnosed multiple myeloma* • in combination with bortezomib, thalidomide and dexamethasone for the treatment of adult patients with newly diagnosed multiple myeloma who are eligible for autologous stem cell transplant • in combination with lenalidomide and dexamethasone, or bortezomib and dexamethasone, for the treatment of adult patients with multiple myeloma who have received at least one prior therapy • in combination with pomalidomide and dexamethasone for the treatment of adult patients with multiple myeloma who have received one prior therapy containing a proteasome inhibitor and lenalidomide and were lenalidomide-refractory, or who have received at least two prior therapies that included lenalidomide and a proteasome inhibitor and have demonstrated disease progression on or after the last therapy (see section 5.1)* • as monotherapy for the treatment of adult patients with relapsed and refractory multiple myeloma, whose prior therapy included a proteasome inhibitor and an immunomodulatory agent and who have demonstrated disease progression on the last therapy • as monotherapy is indicated for the treatment of adult patients with smouldering multiple myeloma at high risk of developing multiple myeloma | PLGB 00242/0676 – 0677/OD1 | 24-May-26 |
| Darzalex | Daratumumab | Systemic light chain (AL) amyloidosis | Darzalex is indicated in combination with cyclophosphamide, bortezomib and dexamethasone for the treatment of adult patients with newly diagnosed systemic light chain (AL) amyloidosis | PLGB 00242/0677/OD2 | 11-Oct-31 |
| Daurismo | glasdegib maleate | Acute myeloid leukaemia | Daurismo is indicated, in combination with low-dose cytarabine, for the treatment of newly diagnosed de novo or secondary acute myeloid leukaemia (AML) in adult patients who are not candidates for standard induction chemotherapy. | PLGB 00057/1687 – 1688/OD1 | 29-Jun-30 |
| Dovprela | pretomanid (S)-2-nitro-6-(4-(trifluoromethoxy)benzyloxy)-6,7-dihydro-5H-imidazo[2,1-b][1,3]oxazine | Tuberculosis | Dovprela is indicated in combination with bedaquiline and linezolid, in adults, for the treatment of pulmonary extensively drug resistant (XDR), or treatment-intolerant or nonresponsive multidrug-resistant (MDR) tuberculosis (TB). | PLGB 46302/0234/OD1 | 04-Aug-30 |
| Ebvallo 2.8 x 10^7 - 7.3 x 10^7 cells/mL dispersion for injection | TABELECLEUCEL | Post transplant Epstein-Barr virus associated lymphoproliferative disorder | treatment of adult and paediatric patients 2 years of age and older with relapsed or refractory Epstein-Barr virus positive post-transplant lymphoproliferative disease (EBV+ PTLD) who have received at least one prior therapy. For solid organ transplant patients, prior therapy includes chemotherapy unless chemotherapy is inappropriate’. | PLGB 52297/0001/OD1 | 22-May-33 |
| Ekterly | Sebetralstat | Hereditary Angioedema | Ekterly is indicated for the treatment of hereditary angioedema (HAE) attacks in adult and adolescents aged 12 years and older. | PL46326/0001/OD1 | 15-Jul-35 |
| ELZONRIS | TAGRAXOFUSP | Blastic plasmacytoid dendritic cell neoplasia | Monotherapy for the first-line treatment of adult patients with blastic plasmacytoid dendritic cell neoplasm | PLGB 53425/0001/OD1 | 14-Oct-31 |
| Enspryng | Satralizumab | Neuromyelitis optica spectrum disorders (NMOSD) | Enspryng is indicated as a monotherapy or in combination with immunosuppressive therapy (IST) for the treatment of neuromyelitis optica spectrum disorders (NMOSD) in adult and adolescent patients from 12 years of age who are anti-aquaporin-4 IgG (AQP4-IgG) seropositive. | PLGB 00031/0916 /OD1 | 21-Nov-33 |
| Epidyolex | cannabidiol | Lennox-Gastaut syndrome | Epidyolex is indicated for use as adjunctive therapy of seizures associated with Lennox Gastaut syndrome (LGS), in conjunction with clobazam, for patients 2 years of age and older. | PLGB 36772/0001/OD1 | 23-Sep-29 |
| Epidyolex | cannabidiol | Dravet syndrome | Epidyolex is indicated for use as adjunctive therapy of seizures associated with Dravet syndrome (DS), in conjunction with clobazam, for patients 2 years of age and older. | PLGB 36772/0001/OD2 | 23-Sep-29 |
| Epidyolex | cannabidiol | Tuberous sclerosis complex | Epidyolex is indicated for use as adjunctive therapy of seizures associated with tuberous sclerosis complex (TSC) for patients 2 years of age and older. | PLGB 36772/0001/OD3 | 05-Aug-31 |
| FABHALTA | IPTACOPAN HYDROCHLORIDE MONOHYDRATE | Paroxysmal Nocturnal Haemoglobinuria (PNH) | Monotherapy in the treatment of adult patients with paroxysmal nocturnal haemoglobinuria (PNH) who have haemolytic anaemia | PLGB 00101/1231/OD1 | 02-Aug-34 |
| FABHALTA???? | IPTACOPAN HYDROCHLORIDE MONOHYDRATE | complement 3 glomerulopathy (C3G) | treatment of adult patients with complement 3 glomerulopathy (C3G) in combination with a renin-angiotensin system (RAS) inhibitor, or in patients who are RAS-inhibitor intolerant, or for whom a RAS inhibitor is contraindicated | PLGB 00101/1231/OD2 | 07-May-35 |
| Filspari | Sparsentan | Primary IGA Nephropathy | Treatment of adults with primary immunoglobulin A nephropathy (IgAN) with a urine protein excretion > 1.0 g/day (or urine protein-to-creatinine ratio ? 0.75 g/g) | PLGB 15240/0005/OD1 - 0006/OD1 | 06-Nov-34 |
| FILSUVEZ | BETULAE CORTEX DRY EXTRACT | Epidermolysis bullosa | Treatment of partial thickness wounds associated with dystrophic and junctional epidermolysis bullosa (EB) in patients 6 months and older | PLGB 50688/0011/OD1 | 11-Aug-32 |
| FINLEE | DABRAFENIB MESYLATE | Glioma | Low-grade glioma Finlee in combination with trametinib is indicated for the treatment of paediatric patients aged 1 year and older with low-grade glioma (LGG) with a BRAF V600E mutation who require systemic therapy. High-grade glioma Finlee in combination with trametinib is indicated for the treatment of paediatric patients aged 1 year and older with high-grade glioma (HGG) with a BRAF V600E mutation who have received at least one prior radiation and/or chemotherapy treatment. | PLGB 00101/1228/OD1 | 20-Mar-36 |
| Fintepla | fenfluramine | Dravet syndrome | Fintepla is indicated for the treatment of seizures associated with Dravet syndrome as an add on therapy to other anti-epileptic medicines for patients 2 years of age and older. | PLGB 45831/0001/OD1 | 21-Dec-30 |
| Fintepla | fenfluramine hydrochloride | Lennox-Gastaut syndrome | Treatment of seizures associated with Lennox- Gastaut syndrome as an add on therapy to other anti-epileptic medicines for patients 2 years of age and older. | PLGB 00039/0804 – 0010OD2 | 05-Jul-35 |
| Galafold | migalastat | Fabry disease | Galafold is indicated for long-term treatment of adults and adolescents aged 16 years and older with a confirmed diagnosis of Fabry disease (?-galactosidase A deficiency) and who have an amenable mutation (see the tables in section 5.1). | PLGB 25823/0002/OD1 | 31-May-26 |
| Gazyvaro | obinutuzumab | Follicular lymphoma (FL) | Gazyvaro in combination with chemotherapy, followed by Gazyvaro maintenance therapy in patients achieving a response, is indicated for the treatment of patients with previously untreated advanced FL (see section 5.1) Gazyvaro in combination with bendamustine followed by Gazyvaro maintenance is indicated for the treatment of patients with FL who did not respond or who progressed during or up to 6 months after treatment with rituximab or a rituximab-containing regimen. | PLGB 00031/0856/OD2 | 15-Jun-26 |
| Gilteritinib Astellas 40mg film-coated tablets | gilteritinib | Acute myeloid leukaemia | Gilteritinib Astellas is indicated as monotherapy for the treatment of adult patients who have relapsed or refractory acute myeloid leukaemia (AML) with a FLT3 mutation. | PLGB 00166/0425/OD1 | 28-Oct-29 |
| Givinostat | GIVINOSTAT HYDROCHLORIDE MONOHYDRATE | Duchenne muscular dystrophy (DMD) | reatment of Duchenne muscular dystrophy (DMD) in patients 6 years of age and older. | PLGB 13297/0017/OD1 | 20-Dec-34 |
| Givlaari | givosiran | Acute hepatic porphyria | Treatment of acute hepatic porphyria (AHP) in adults and adolescents aged 12 years and older | PLGB 43942/0001/OD1 | 04-Mar-30 |
| Hemgenix 1 x 10^13 genome copies/mL concentrate for solution for infusion | ETRANACOGENE DEZAPARVOVEC | Haemophilia B (Factor IX) | treatment of severe and moderately severe Haemophilia B (congenital Factor IX deficiency) in adult patients without a history of Factor IX inhibitors. | PLGB 15036/0160/OD1 | 22-Mar-33 |
| Holoclar | Ex-vivo expanded autologous human corneal epithelium containing stem cells | Corneal lesions, with associated corneal (limbal) stem cell deficiency, due to ocular burns | Treatment of adult patients with moderate to severe limbal stem cell deficiency (defined by the presence of superficial corneal neovascularisation in at least two corneal quadrants, with central corneal involvement, and severely impaired visual acuity), unilateral or bilateral, due to physical or chemical ocular burns. A minimum of 1-2 mm2 of undamaged limbus is required for biopsy. | PLGB 58508/0001 - 0003 | 19-Feb-27 |
| Hyftor | SIROLIMUS | Tuberous sclerosis | Hyftor is indicated for the treatment of facial angiofibroma associated with tuberous sclerosis complex in adults and paediatric patients aged 6 years and older | PLGB 57572/0001/OD1 | 01-Sep-33 |
| Idefirix | imlifidase Recombinant IgG degrading enzyme of Streptococcus pyogenes | Graft rejection following solid organ transplantation | Idefirix is indicated for desensitisation treatment of highly sensitised adult kidney transplant patients with positive crossmatch against an available deceased donor. The use of Idefirix should be reserved for patients unlikely to be transplanted under the available kidney allocation system including prioritisation programmes for highly sensitised patients. | PLGB 46323/0002/OD1 | 01-Sep-30 |
| IDELVION | albutrepenonacog alfa Recombinant fusion protein linking human coagulation factor IX with human albumin | Haemophilia B | Treatment and prophylaxis of bleeding in patients with haemophilia B (congenital factor IX deficiency). | PLGB 00231/0337 – 0340/OD1 | 13-May-26 |
| Imcivree | setmelanotide | Genetic obesity disorders caused by defects in melanocortin 4 receptor (MC4R) pathway, including POMC and LEPR deficiency obesity | The treatment of obesity and the control of hunger associated with genetically confirmed loss of function biallelic pro-opiomelanocortin (POMC), including PCSK1 deficiency or biallelic leptin receptor (LEPR) deficiency in adults and children 2 years of age and above | PLGB 55587/0001/OD1 | 14-Sep-31 |
| Imcivree | setmelanotide | Bardet-Biedl syndrome (BBS) | The treatment of obesity and the control of hunger associated with genetically confirmed Bardet-Biedl syndrome (BBS) in adults and children 2 years of age and above | PLGB 55587/0001OD2 | 17-Nov-32 |
| IMDYLLTRA 1 milligram and 10mg powder for concentrate and solution for infusion | TARLATAMAB | Small cell lung cancer | For the treatment of adult patients with extensive-stage small cell lung cancer (ES-SCLC) with disease progression on or after at least two prior lines of therapy including platinum-based chemotherapy | PLGB 13832/0083-0084/OD1 | 31-Dec-34 |
| Inrebic | fedratinib dihydrochloride monohydrate | Myelofibrosis | Inrebic is indicated for the treatment of disease-related splenomegaly or symptoms in adult patients with primary myelofibrosis, post polycythaemia vera myelofibrosis or post essential thrombocythaemia myelofibrosis who are Janus Associated Kinase (JAK) inhibitor naïve or have been treated with ruxolitinib. | PLGB 50412/0022/OD1 | 16-Apr-31 |
| IQIRVO | Elafibranor | Primary Biliary Cholangitis | Iqirvo is indicated for the treatment of primary biliary cholangitis. | PLGB 34926/0029/OD1 | 01-Oct-34 |
| Isturisa | osilodrostat | Cushing’s syndrome | Isturisa is indicated for the treatment of endogenous Cushing’s syndrome in adults. | PLGB 15266/0029 – 0031/OD1 | 13-Jan-30 |
| Ixazomib | ixazomib 2,2’-{2-[(1R)-1-({[(2,5-dichlorobenzoyl)amino]acetyl}amino)-3-methylbutyl]-5-oxo-1,3,2-dioxaborolane-4,4-diyl}diacetic acid | Multiple myeloma | NINLARO in combination with lenalidomide and dexamethasone is indicated for the treatment of adult patients with multiple myeloma who have received at least one prior therapy. | PLGB 16189/0109 – 0111/OD1 | 23-Nov-26 |
| Joenja | LENIOLISIB | Activated phosphoinositide 3 kinase delta (P13K?) syndrome (APDS) | Joenja is indicated for the treatment of activated phosphoinositide 3-kinase delta (P13K?) syndrome (APDS) in adult and paediatric patients 12 years of age and older. | PLGB 33010/0001/OD1 | 25-Sep-34 |
| Jorveza | budesonide | Eosinophilic oesophagitis | Jorveza is indicated for the treatment of eosinophilic esophagitis (EoE) in adults (older than 18 years of age). | PLGB 08637/0030-0032/OD1 | 10-Jan-28 |
| Kaftrio | ivacaftor, tezacaftor, N-(1,3-dimethyl-1H-pyrazole-4-sulfonyl)-6-[3-(3,3,3-trifluoro-2,2-dimethylpropoxy)-1H-pyrazol-1-yl]-2-[(4S)-2,2,4-trimethylpyrrolidin-1-yl]pyridine-3-carboxamide (elexacaftor) | Cystic fibrosis | Kaftrio is indicated in a combination regimen with ivacaftor for the treatment of cystic fibrosis (CF) in patients aged 6 years and older who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene (see section 5.1). | PLGB 22352/0012/OD1 & PLGB 22352/0017/OD1 | 21-Aug-30 |
| KIMMTRAK | tebentafusp | Uveal melanoma | KIMMTRAK is indicated as monotherapy for the treatment of HLA-A*02:01-positive adult patients with unresectable or metastatic uveal melanoma. | PLGB 36781/0001/OD1 | 06-Jun-32 |
| Kinpeygo | Budesonide | Primary immunoglobulin A (IgA) nephropathy | Treatment of primary immunoglobulin A (IgA) nephropathy (IgAN) in adults at risk of rapid disease progression with a urine protein-to-creatinine ratio (UPCR) ?1.5 g/gram | PLGB 49329/0001/OD1 | 01-Feb-33 |
| Koselugo | selumetinib hydrogen sulfate | Neurofibromatosis Type 1 | Treatment of symptomatic, inoperable plexiform neurofibromas (PN) in paediatric patients with neurofibromatosis type 1 (NF1) aged 3 years and above. | PLGB 17901/0356 - 0357/OD1 | 08-Aug-31 |
| Kymriah | tisagenlecleucel Autologous T cells transduced with lentiviral vector containing a chimeric antigen receptor directed against CD19 | B-lymphoblastic leukaemia | Kymriah is indicated for the treatment of paediatric and young adult patients up to 25 years of age with B cell acute lymphoblastic leukaemia (ALL) that is refractory, in relapse post transplant or in second or later relapse. | PL 08498/0039/OD1 | 27-Aug-28 |
| Kymriah | tisagenlecleucel Autologous T cells transduced with lentiviral vector containing a chimeric antigen receptor directed against CD19 | Diffuse large B-cell lymphoma | Kymriah is indicated for the treatment of: Adult patients with relapsed or refractory diffuse large B cell lymphoma (DLBCL) after two or more lines of systemic therapy. | PLGB 00101/1101/OD2 | 27-Aug-28 |
| Kymriah | tisagenlecleucel Autologous T cells transduced with lentiviral vector containing a chimeric antigen receptor directed against CD19 | Follicular lymphoma | Treatment of adult patients with relapsed or refractory follicular lymphoma (FL) after two or more lines of systemic therapy | PLGB 00101/1101/OD3 | 09-Aug-32 |
| Kyprolis | carfilzomib | Multiple myeloma | Kyprolis in combination with daratumumab and dexamethasone, with lenalidomide and dexamethasone, or with dexamethasone alone is indicated for the treatment of adult patients with multiple myeloma who have received at least one prior therapy. | PLGB 13832/0023 – 0025/OD1 | 23-Nov-25 |
| Lamzede | velmanase alfa Recombinant human ?-Mannosidase | ?-Mannosidosis | Enzyme replacement therapy for the treatment of non-neurological manifestations in patients with mild to moderate alpha mannosidosis. See sections 4.4 and 5.1. | PLGB 08829/0188/OD1 | 27-Mar-28 |
| Ledaga | chlormethine | Cutaneous T-cell lymphoma | Ledaga is indicated for the topical treatment of mycosis fungoides-type cutaneous T-cell lymphoma (MF type CTCL) in adult patients | PLGB 15720/0005/OD1 | 07-Mar-27 |
| Libmeldy | Autologous CD34+ cell enriched population that contains hematopoietic stem and progenitor cells transduced ex vivo using a lentiviral vector encoding the human arylsulfatase A gene | Metachromatic leukodystrophy | ibmeldy is indicated for the treatment of metachromatic leukodystrophy (MLD) characterized by biallelic mutations in the arysulfatase A (ARSA) gene leading to a reduction of the ARSA enzymatic activity: in children with late infantile or early juvenile forms, without clinical manifestations of the disease, in children with the early juvenile form, with early clinical manifestations of the disease, who still have the ability to walk independently and before the onset of cognitive decline | PLGB 49055/0002/OD1 | 18-Dec-30 |
| LIVMARLI | maralixibat chloride | Cholestatic pruritus | Treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) 2 months of age and older | PLGB 56642/0001/OD1 | 10-Feb-35 |
| LIVTENCITY | maribavir | Cytomegalovirus (CMV) infection in patients with impaired cell-mediated immunity | Treatment of cytomegalovirus (CMV) infection and/or disease that are refractory (with or without resistance) to one or more prior therapies, including ganciclovir, valganciclovir, cidofovir or foscarnet in adult patients who have undergone a haematopoietic stem cell transplant (HSCT) or solid organ transplant (SOT). Consideration should be given to official guidance on the appropriate use of antiviral agents. | PLGB 16189/0127/OD1 | 11-Nov-32 |
| LIVTENCITY | maribavir | Cytomegalovirus (CMV) disease in patients with impaired cell-mediated immunity | Treatment of cytomegalovirus (CMV) infection and/or disease that are refractory (with or without resistance) to one or more prior therapies, including ganciclovir, valganciclovir, cidofovir or foscarnet in adult patients who have undergone a haematopoietic stem cell transplant (HSCT) or solid organ transplant (SOT). Consideration should be given to official guidance on the appropriate use of antiviral agents | PLGB 16189/0127/OD2 | 11-Nov-32 |
| Loargys 5 mg/ml solution for injection/infusion | PEGZILARGINASE | Arginase 1 deficiency | Treatment of arginase 1 deficiency (ARG1-D), also known as hyperargininemia, in adults, adolescents and children aged 2 years and older. | PLGB 53487/0007/OD1 | 20-Dec-33 |
| Lonapegsomatropin Ascendis Pharma | lonapegsomatropin | Growth hormone deficiency | Growth failure in children and adolescents aged from 3 years up to 18 years due to insufficient endogenous growth hormone secretion (growth hormone deficiency [GHD]) | PLGB 47497/0001-009/OD1 | 17-Oct-32 |
| LUNSUMIO | mosunetuzumab | Follicular lymphoma | Treatment of adult patients with relapsed or refractory follicular lymphoma (FL) who have received at least two prior systemic therapies | PLGB 00031/0933-0934/OD1 | 04-Oct-32 |
| Lutathera | lutetium (177Lu) oxodotreotide Lutetium (177Lu)-N-[(4,7,10-Tricarboxymethyl-1,4,7,10-tetraazacyclododec-1-yl)acetyl]-D-phenylalanyl-L-cysteinyl-L-tyrosyl-D-tryptophanyl-L-lysyl-L-threoninyl | Gastro-entero-pancreatic neuroendocrine tumours | Lutathera is indicated for the treatment of unresectable or metastatic, progressive, well differentiated (G1 and G2), somatostatin receptor positive gastroenteropancreatic neuroendocrine tumours (GEP NETs) in adults. | PLGB 35145/0003/OD1 | 28-Sep-27 |
| Luxturna | voretigene neparvovec | Inherited retinal dystrophies (initially named Leber’s congenital amaurosis) | Luxturna is indicated for the treatment of adult and paediatric patients with vision loss due to inherited retinal dystrophy caused by confirmed biallelic RPE65 mutations and who have sufficient viable retinal cells. | PLGB 00101/1104/OD1 | 05-Dec-28 |
| Luxturna | voretigene neparvovec | Inherited retinal dystrophies (initially named retinitis pigmentosa) | Luxturna is indicated for the treatment of adult and paediatric patients with vision loss due to inherited retinal dystrophy caused by confirmed biallelic RPE65 mutations and who have sufficient viable retinal cells. | PLGB 00101/1104/OD2 | 05-Dec-28 |
| Mepsevil | vestronidase alfa Recombinant human beta-glucuronidase | Mucopolysaccharidosis type VII (Sly syndrome) | Mepsevil is indicated for the treatment of non-neurological manifestations of Mucopolysaccharidosis VII (MPS VII; Sly syndrome). | PLGB 474793/0002/OD1 | 27-Aug-28 |
| Minjuvi | tafasitamab | Relapsed or refractory diffuse large B-cell lymphoma | Minjuvi is indicated in combination with lenalidomide followed by Minjuvi monotherapy for the treatment of adult patients with relapsed or refractory diffuse large B cell lymphoma (DLBCL) who are not eligible for autologous stem cell transplant (ASCT). | PLGB 42338/0016/OD1 | 07-Oct-31 |
| Myalepta | metreleptin | Familial Partial Lipodystrophy | Myalepta is indicated as an adjunct to diet as a replacement therapy to treat the complications of leptin deficiency in lipodystrophy (LD) patients: with confirmed familial partial LD in adults and children 12 years of age and above for whom standard treatments have failed to achieve adequate metabolic control. | PLGB 50688/0008-0010/OD1 | 01-Aug-28 |
| Myalepta | metreleptin | Barraquer-Simons syndrome | Myalepta is indicated as an adjunct to diet as a replacement therapy to treat the complications of leptin deficiency in lipodystrophy (LD) patients: with acquired partial LD (Barraquer-Simons syndrome), in adults and children 12 years of age and above for whom standard treatments have failed to achieve adequate metabolic control. | PLGB 50688/0008-0010/OD2 | 01-Aug-28 |
| Myalepta | metreleptin | Lawrence syndrome | Myalepta is indicated as an adjunct to diet as a replacement therapy to treat the complications of leptin deficiency in lipodystrophy (LD) patients: with acquired generalised LD (Lawrence syndrome) in adults and children 2 years of age and above | PLGB 50688/0008-0010/OD3 | 01-Aug-28 |
| Myalepta | metreleptin | Berardinelli-Seip syndrome | Myalepta is indicated as an adjunct to diet as a replacement therapy to treat the complications of leptin deficiency in lipodystrophy (LD) patients: with confirmed congenital generalised LD (Berardinelli-Seip syndrome) | PLGB 50688/0008-0010/OD4 | 01-Aug-28 |
| Mylotarg | gemtuzumab ozogamicin | Acute myeloid leukaemia | MYLOTARG is indicated for combination therapy with daunorubicin (DNR) and cytarabine (AraC) for the treatment of patients age 15 years and above with previously untreated, de novo CD33 positive acute myeloid leukaemia (AML), except acute promyelocytic leukaemia (APL). | PLGB 00057/1591/OD1 | 23-Apr-28 |
| Namuscla | mexiletine hydrochloride | Myotonic disorders | Namuscla is indicated for the symptomatic treatment of myotonia in adult patients with non-dystrophic myotonic disorders | PLGB 35507/0200/OD1 | 20-Dec-28 |
| Natpar | parathyroid hormone Recombinant human parathyroid hormone | Hypoparathyroidism | Natpar is indicated as adjunctive treatment of adult patients with chronic hypoparathyroidism who cannot be adequately controlled with standard therapy alone. | PLGB 16189/0067 – 0070/OD1 | 26-Apr-27 |
| Nexviadyme 100 mg powder for concentrate for solution for infusion | AVALGLUCOSIDASE ALFA | Glycogen storage disease type II (Pompe’s disease) | Long-term enzyme replacement therapy for the treatment of patients with Pompe disease (acid ?-glucosidase deficiency) | PLGB 04425/0893/OD1 | 03-May-33 |
| Ngenla | somatrogon | Growth hormone deficiency | Ngenla is indicated for the treatment of children and adolescents from 3 years of age with growth disturbance due to insufficient secretion of growth hormone. | PLGB 00057/1712-1713/OD1 | 25-Mar-32 |
| Ocaliva | obeticholic acid 6alpha-ethyl-chenodeoxycholic acid | Primary biliary cirrhosis | Ocaliva is indicated for the treatment of primary biliary cholangitis (PCB) (also known as primary biliary cirrhosis) in combination with ursodeoxycholic acid (UDCA) in adults with an inadequate response to UDCA or as monotherapy in adults unable to tolerate UDCA. | PLGB 48025/0002 – 0003/OD1 | 15-Dec-26 |
| Onivyde pegylated liposomal | irinotecan Nanoliposomal irinotecan | Pancreatic cancer | In combination with oxaliplatin, 5?fluorouracil (5?FU) and leucovorin (LV) for the first-line treatment of adult patients with metastatic adenocarcinoma of the pancreas, in combination with 5-FU and LV for the treatment of metastatic adenocarcinoma of the pancreas, in combination with 5?fluorouracil (5?FU) and leucovorin (LV), in adult patients who have progressed following gemcitabine based therapy. | PLGB 05815/0111/OD1 | 18-Oct-26 |
| Onpattro | patisiran Synthetic double-stranded siRNA oligonucleotide directed against transthyretin mRNA | Transthyretin-mediated amyloidosis | Onpattro is indicated for the treatment of hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) in adult patients with stage 1 or stage 2 polyneuropathy. | PLGB 50597/0002/OD1 | 29-Aug-28 |
| Opfolda | MIGLUSTAT | Pompe’s disease adult onset | Opfolda (miglustat) is an enzyme stabiliser of cipaglucosidase alfa long-term enzyme replacement therapy in adults with late-onset Pompe disease (acid ??glucosidase [GAA] deficiency). | PLGB 25823/0004/OD1 | 08-Aug-33 |
| OXBRYTA | voxelotor | Sickle cell disease | Oxbryta is indicated for the treatment of haemolytic anaemia due to sickle cell disease (SCD) in adults and paediatric patients 12 years of age and older as monotherapy or in combination with hydroxycarbamide. | PLGB 54981/0001/OD1 | 25-Jul-32 |
| Oxlumo | lumasiran Synthetic double-stranded siRNA oligonucleotide directed against hydroxyacid oxidase 1 mRNA and covalently linked to a ligand containing three N-acetylgalactosamine residues | Primary hyperoxaluria | Oxlumo is indicated for the treatment of primary hyperoxaluria type 1 (PH1) in all age groups. | PLGB 43942/0003/OD1 | 23-Nov-30 |
| Pemazyre | pemigatinib | Biliary tract cancer | The treatment of adults with locally advanced or metastatic cholangiocarcinoma with a fibroblast growth factor receptor 2 (FGFR2) fusion or rearrangement that have progressed after at least one prior line of systemic therapy. | PLGB 42338/0008 - 0010/OD1 | 07-Apr-31 |
| Polivy | polatuzumab vedotin | Diffuse large B-cell lymphoma | Polivy in combination with rituximab, cyclophosphamide, doxorubicin, and prednisone (R-CHP) is indicated for the treatment of adult patients with previously untreated diffuse large B-cell lymphoma (DLBCL). Polivy in combination with bendamustine and rituximab is indicated for the treatment of adult patients with relapsed/refractory diffuse large B-cell lymphoma (DLBCL) who are not candidates for haematopoietic stem cell transplant. | PLGB 00031/0912/OD1 | 20-Jan-30 |
| Pombiliti 105 mg powder for concentrate for solution for infusion | CIPAGLUCOSIDASE ALFA | Glycogen storage disease type II (Pompe’s disease) | Authorised orphan indication: Long-term enzyme replacement therapy used in combination with the enzyme stabiliser miglustat for the treatment of adults with late-onset Pompe disease (acid ?-glucosidase [GAA] deficiency | PLGB 25823/0003/OD1 | 30-Jun-33 |
| Poteligeo | mogamulizumab | Cutaneous T-cell lymphoma | POTELIGEO is indicated for the treatment of adult patients with mycosis fungoides (MF) or Sézary syndrome (SS) who have received at least one prior systemic therapy. | PLGB 50262/0008/OD1 | 26-Nov-28 |
| PREVYMIS | letermovir (S)-{8-fluoro-2-2[4-(3-methoxyphenyl)-1-piperazinyl]-3-[2-methoxy-5-(trifluoromethyl)-phenyl]-3,4-dihydro-4-quinazolinyl} acetic acid | Cytomegalovirus disease reactivation in patients with impaired cell-mediated immunity | PREVYMIS is indicated for prophylaxis of cytomegalovirus (CMV) reactivation and disease in adult CMV-seropositive recipients [R+] of an allogeneic haematopoietic stem cell transplant (HSCT). Consideration should be given to official guidance on the appropriate use of antiviral agents. | PLGB 53095/0046 – 0048/OD1 | 10-Jan-28 |
| PYRUKYND | Mitapivat sulfate Potent, allosteric activator of wild-type red blood cell (RBC)-specific form of pyruvate kinase (PKR) and a range of mutant PKR enzymes. Mitapivat targets the underlying enzymatic defect that causes haemolysis in pyruvate kinase deficiency by restoring the activity of mutant forms of PKR. | pyruvate kinase deficiency (PK deficiency) | Pyrukynd is indicated for the treatment of pyruvate kinase deficiency (PK deficiency) in adult patients | PLGB 52779/0001-0005OD1 | 29-Nov-32 |
| Qalsody | Tofersen | Amyotrophic lateral sclerosis (ALS) | Treatment of adults with amyotrophic lateral sclerosis (ALS), associated with a mutation in the superoxide dismutase 1 (SOD1) gene | PL 22407/0034/OD1 | 22 July 2035 |
| Qarziba | dinutuximab beta Chimeric monoclonal antibody against GD2 | Neuroblastoma | Qarziba is indicated for the treatment of high-risk neuroblastoma in patients aged 12 months and above, who have previously received induction chemotherapy and achieved at least a partial response, followed by myeloablative therapy and stem cell transplantation, as well as patients with history of relapsed or refractory neuroblastoma, with or without residual disease. Prior to the treatment of relapsed neuroblastoma, any actively progressing disease should be stabilised by other suitable measures. In patients with a history of relapsed/refractory disease and in patients who have not achieved a complete response after first line therapy, Qarziba should be combined with interleukin-2 (IL-2). | PLGB 44185/0005/OD1 | 11-May-27 |
| QINLOCK | ripretinib | Gastrointestinal stromal tumours | QINLOCK is indicated for the treatment of adult patients with advanced gastrointestinal stromal tumour (GIST) who have received prior treatment with three or more kinase inhibitors, including imatinib | PLGB 55295/0004/OD1 | 20-Dec-31 |
| Ravicti | Glycerol phenylbutyrate Glyceryl tri-(4-phenylbutyrate) | Carbamoyl-phosphate synthase-1 deficiency | RAVICTI is indicated for use as adjunctive therapy for chronic management of patients with urea cycle disorders (UCDs) who cannot be managed by dietary protein restriction and/or amino acid supplementation alone. RAVICTI must be used with dietary protein restriction and, in some cases, dietary supplements (e.g., essential amino acids, arginine, citrulline, protein-free calorie supplements). | PLGB 53487/0001/OD1 | 01-Dec-27 |
| Ravicti | Glycerol phenylbutyrate Glyceryl tri-(4-phenylbutyrate) | Ornithine carbamoyltransferase deficiency | RAVICTI is indicated for use as adjunctive therapy for chronic management of patients with urea cycle disorders (UCDs) who cannot be managed by dietary protein restriction and/or amino acid supplementation alone. RAVICTI must be used with dietary protein restriction and, in some cases, dietary supplements (e.g., essential amino acids, arginine, citrulline, protein-free calorie supplements). | PLGB 53487/0001/OD2 | 01-Dec-27 |
| Ravicti | Glycerol phenylbutyrate Glyceryl tri-(4-phenylbutyrate) | Citrullinaemia type 1 | RAVICTI is indicated for use as adjunctive therapy for chronic management of patients with urea cycle disorders (UCDs) who cannot be managed by dietary protein restriction and/or amino acid supplementation alone. RAVICTI must be used with dietary protein restriction and, in some cases, dietary supplements (e.g., essential amino acids, arginine, citrulline, protein-free calorie supplements). | PLGB 53487/0001/OD3 | 01-Dec-27 |
| Ravicti | Glycerol phenylbutyrate Glyceryl tri-(4-phenylbutyrate) | Argininosuccinic aciduria | RAVICTI is indicated for use as adjunctive therapy for chronic management of patients with urea cycle disorders (UCDs) who cannot be managed by dietary protein restriction and/or amino acid supplementation alone. RAVICTI must be used with dietary protein restriction and, in some cases, dietary supplements (e.g., essential amino acids, arginine, citrulline, protein-free calorie supplements). | PLGB 53487/0001/OD4 | 01-Dec-27 |
| Ravicti | Glycerol phenylbutyrate Glyceryl tri-(4-phenylbutyrate) | Hyperargininaemia | RAVICTI is indicated for use as adjunctive therapy for chronic management of patients with urea cycle disorders (UCDs) who cannot be managed by dietary protein restriction and/or amino acid supplementation alone. RAVICTI must be used with dietary protein restriction and, in some cases, dietary supplements (e.g., essential amino acids, arginine, citrulline, protein-free calorie supplements). | PLGB 53487/0001/OD5 | 01-Dec-27 |
| Ravicti | Glycerol phenylbutyrate Glyceryl tri-(4-phenylbutyrate) | Ornithine translocase deficiency (hyperornithinaemia-hyperammonaemia homocitrullinuria (HHH) syndrome) | Treatment of ornithine translocase deficiency (hyperornithinaemia-hyperammonaemia homocitrullinuria (HHH) syndrome): RAVICTI is indicated for use as adjunctive therapy for chronic management of patients with urea cycle disorders (UCDs) who cannot be managed by dietary protein restriction and/or amino acid supplementation alone. RAVICTI must be used with dietary protein restriction and, in some cases, dietary supplements (e.g., essential amino acids, arginine, citrulline, protein-free calorie supplements). | PLGB 53487/0001/OD6 | 01-Dec-27 |
| Reblozyl | luspatercept Recombinant fusion protein consisting of a modified form of the extracellular domain of human activin receptor IIB linked to the human IgG1 Fc domain | Myelodysplastic syndromes | In adults for the treatment of transfusion-dependent anaemia due to very low, low and intermediate-risk myelodysplastic syndromes (MDS). | PLGB 15105/0153 - 1054/OD2 | 26-Jun-30 |
| REZUROCK | belumosudil mesilate | Graft versus host disease (GVHD) | Rezurock is indicated for the treatment of patients aged 12 years and older with chronic graft-versus-host disease (chronic GVHD) who have received at least two prior lines of systemic therapy. | PLGB 53904/0001/OD1 | 06-Jul-32 |
| Rezzayo | REZAFUNGIN ACETATE | Treatment of invasive candidiasis | Treatment of invasive candidiasis in adult | PLGB 16950/0390/OD1 | 29/01/2034 |
| Rydapt | midostaurin | Acute myeloid leukaemia | Rydapt is indicated in combination with standard daunorubicin and cytarabine induction and high dose cytarabine consolidation chemotherapy, and for patients in complete response followed by Rydapt single agent maintenance therapy, for adult patients with newly diagnosed acute myeloid leukaemia (AML) who are FLT3 mutation positive | PLGB 00101/1130/OD1 | 20-Sep-27 |
| Rydapt | midostaurin | Mastocytosis | Rydapt is indicated as monotherapy for the treatment of adult patients with aggressive systemic mastocytosis (ASM), systemic mastocytosis with associated haematological neoplasm (SM AHN), or mast cell leukaemia (MCL). | PLGB 00101/1130/OD2 | 20-Sep-27 |
| Rystiggo 140 mg/ml solution for injection | ROZANOLIXIZUMAB | Myasthenia gravis | as an add-on to standard therapy for the treatment of generalised myasthenia gravis (gMG) in adult patients who are anti-acetylcholine receptor (AChR) or anti-muscle-specific tyrosine kinase (MuSK) antibody positive. | PLGB 00039/0808/OD1 | 07/03/2034 |
| Scemblix | Asciminib | Chronic myeloid leukaemia | Scemblix is indicated for the treatment of adult patients with Philadelphia chromosome-positive chronic myeloid leukaemia in chronic phase, previously treated with two or more tyrosine kinase inhibitors, and without a known T315I mutation | PLGB 00101/1207/OD1 | 15-Jun-32 |
| Skyclarys 50 mg hard capsules | OMAVELOXOLONE | Friedreich’s ataxia | treatment of Friedreich’s ataxia in adults and adolescents aged 16 years and older. | PLGB 22407/0033/OD1 | 23-Apr-35 |
| Sogroya | somapacitan | Growth hormone deficiency | Sogroya is indicated for the replacement of endogenous growth hormone (GH) in children aged 3 years and above, and adolescents with growth failure due to growth hormone deficiency (paediatric GHD), and in adults with growth hormone deficiency (adult GHD). | PLGB 04668/0434/OD1 | 21-Oct-31 |
| Soliris | eculizumab | Myasthenia gravis | Soliris is indicated for the treatment of refractory generalized myasthenia gravis (gMG) in patients aged 6 years and above who are antiacetylcholine receptor (AChR) antibody-positive | PLGB 31775/0003/OD2 | 17-Aug-27 |
| Soliris | eculizumab | Neuromyelitis optica spectrum disorders | Soliris is indicated in adults for the treatment of neuromyelitis optica spectrum disorder (NMOSD) in patients who are anti-aquaporin-4 (AQP4) antibody-positive with a relapsing course of the disease. | PLGB 31775/0003/OD3 | 28-Aug-29 |
| SomaKit TOC | edotreotide Gallium (68Ga)-edotreotide | Gastro-entero-pancreatic neuroendocrine tumours | After radiolabelling with gallium (68Ga) chloride solution, the solution of gallium (68Ga) edotreotide obtained is indicated for Positron Emission Tomography (PET) imaging of somatostatin receptor overexpression in adult patients with confirmed or suspected well-differentiated gastro-enteropancreatic neuroendocrine tumours (GEP-NET) for localizing primary tumours and their metastases. | PLGB 35145/0004/OD1 | 12-Dec-26 |
| Spexotras | Trametinib | Glioma | Low-grade Glioma: Spexotras in combination with dabrafenib is indicated for the treatment of paediatric patients aged 1 year and older with low-grade glioma (LGG) with a BRAF V600E mutation who require systemic therapy. High-grade glioma Spexotras in combination with dabrafenib is indicated for the treatment of paediatric patients aged 1 year and older with high-grade glioma (HGG) with a BRAF V600E mutation who have received at least one prior radiation and/or chemotherapy treatment. | PLGB 00101/1227/OD1 | 13-Feb-36 |
| Spinraza | nusinersen Antisense oligonucleotide targeted to the SMN2 gene | 5q spinal muscular atrophy | Spinraza is indicated for the treatment of 5q Spinal Muscular Atrophy. | PLGB 22407/0018/OD1 | 01-Jun-29 |
| Strimvelis | autologous CD34+ enriched cell fraction that contains CD34+ cells transduced with retroviral vector that encodes for the human ADA cDNA sequence | Severe combined immunodeficiency (SCID) due to adenosine deaminase (ADA) deficiency | Strimvelis is indicated for the treatment of patients with severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID), for whom no suitable human leukocyte antigen (HLA)-matched related stem cell donor is available. | PLGB 49055/0001/OD1 | 30-May-28 |
| Symkevi | tezacaftor/ivacaftor | Cystic fibrosis | Symkevi is indicated in a combination regimen with ivacaftor tablets for the treatment of patients with cystic fibrosis (CF) aged 6 years and older who are homozygous for the F508del mutation or who are heterozygous for the F508del mutation and have one of the following mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene: P67L, R117C, L206W, R352Q, A455E, D579G, 711+3A?G, S945L, S977F, R1070W, D1152H, 2789+5G?A, 3272 26A?G, and 3849+10kbC?T. | PLGB 22352/0003/OD1 | 06-Nov-28 |
| TAKHZYRO | lanadelumab Recombinant human IgG1 kappa light chain monoclonal antibody targeting plasma kallikrein | Hereditary angioedema | TAKHZYRO is indicated for routine prevention of recurrent attacks of hereditary angioedema (HAE) in patients aged 2 years and older. | PLGB 54937/0017/OD1, PLGB 54937/0020/OD1, PLGB 54937/0027/OD1, PLGB 54937/0028/OD1’ | 26-Nov-30 |
| Tecartus | autologous anti-CD19-transduced CD3+ cells | Acute lymphoblastic leukaemia | Tecartus is indicated for the treatment of adult patients 26 years of age and above with relapsed or refractory B-cell precursor acute lymphoblastic leukaemia (ALL). | PLGB 11972/0045/OD2 | 07-Nov-32 |
| Tecartus | autologous anti-CD19-transduced CD3+ cells | Mantle cell lymphoma | Tecartus is indicated for the treatment of adult patients with relapsed or refractory mantle cell lymphoma (MCL) after two or more lines of systemic therapy including a Bruton’s tyrosine kinase (BTK) inhibitor. | PLGB 11972/0045/OD1 | 14-Dec-30 |
| Tegsedi | inotersen | ATTR amyloidosis | Tegsedi is indicated for the treatment of stage 1 or stage 2 polyneuropathy in adult patients with hereditary transthyretin amyloidosis (hATTR). | PLGB 51704/0002/OD1 | 10-Jul-28 |
| TIBSOVO | ivosidenib | Acute myeloid leukaemia | In combination with azacitidine for the treatment of adult patients with newly diagnosed acute myeloid leukaemia (AML) with an isocitrate dehydrogenase-1 (IDH1) R132 mutation who are not eligible to receive standard induction chemotherapy (see section 5.1) | PLGB 05815/0120/OD1 | 05-Jul-33 |
| TIBSOVO | ivosidenib | Biliary tract cancer | as monotherapy for the treatment of adult patients with locally advanced or metastatic cholangiocarcinoma with an IDH1 R132 mutation who were previously treated by at least one prior line of systemic therapy (see section 5.1). | PLGB 05815/0120/OD2 | 05-Jul-33 |
| Trecondi | treosulfan | Haematopoietic progenitor cell transplantation | Treosulfan in combination with fludarabine is indicated as part of conditioning treatment prior to allogeneic haematopoietic stem cell transplantation (alloHSCT) in adult patients and in paediatric patients older than one month with malignant and non malignant diseases. | PLGB 11587/0118 - 0119/OD1 | 24-Jun-31 |
| Trepulmix | treprostinil sodium | Chronic thromboembolic pulmonary hypertension | Trepulmix is indicated for the treatment of adult patients with WHO Functional Class (FC) III or IV and: inoperable chronic thromboembolic pulmonary hypertension (CTEPH), or persistent or recurrent CTEPH after surgical treatment to improve exercise capacity. | PLGB 51174/0001 – 0004/OD1 | 07-Apr-30 |
| UPSTAZA | eladocagene exuparvovec | Aromatic L-amino acid decarboxylase deficiency | For the treatment of patients aged 18 months and older with a clinical, molecular, and genetically confirmed diagnosis of aromatic L-amino acid decarboxylase (AADC) deficiency with a severe phenotype. | PLGB 44221/0006/OD1V | 17-Nov-34 |
| Vanflyta | Quizartinib | Acute myeloid leukaemia | In combination with standard cytarabine and anthracycline induction and standard cytarabine consolidation chemotherapy, followed by VANFLYTA single-agent maintenance therapy for adult patients with newly diagnosed acute myeloid leukaemia (AML) that is FLT3-ITD positive | PLGB 08265/0047-0048/OD1 | 11-Mar-34 |
| Verkazia | ciclosporin | Vernal keratoconjunctivitis | Treatment of severe vernal keratoconjunctivitis (VKC) in children from 4 years of age and adolescents | PLGB 16508/0028/OD1 | 10-Jul-30 |
| Voraxaze 1000 units powder for solution for injection | GLUCARPIDASE | Methotrexate-induced nephropathy | Voraxaze is indicated to reduce toxic plasma methotrexate concentration in adults and children (aged 28 days and older) with delayed methotrexate elimination. | PLGB 18442/0002/OD1 | 15-Jun-35 |
| Vyloy | zolbetuximab | Malignant neoplasm of stomach | Vyloy, in combination with fluoropyrimidine- and platinum-containing chemotherapy, is indicated for the first-line treatment of adult patients with locally advanced unresectable or metastatic human epidermal growth factor receptor 2 -negative gastric or gastro-oesophageal junction adenocarcinoma whose tumours are Claudin 18.2 positive | PLGB 00166/0439/OD1 | 14-Aug-34 |
| Vyndaqel | tafamidis N-methyl D-(2,3,4,5,6-pentahydroxy-hexyl)-ammonium; 2-(3,5-dichloro-phenyl)-benzoxazole-6-carboxylate | Senile systemic amyloidosis | Treatment of wild-type transthyretin amyloidosis in adult patients with cardiomyopathy | PLGB 00057/1660/OD2 | 19-Feb-30 |
| Vyvgart 20 mg/ml Concentrate for solution for infusion | EFGARTIGIMOD ALFA | Myasthenia Gravis (gMG) | Treatment of adult patients with generalised Myasthenia Gravis (gMG) who are anti-acetylcholine receptor (AChR) antibody positive. | PLGB 47104/0004/OD1 | 14-Mar-33 |
| Vyxeos liposomal | daunorubicin / cytarabine Liposomal combination of cytarabine and daunorubicin | Acute myeloid leukaemia | Vyxeos is indicated for the treatment of adults with newly diagnosed, therapy-related acute myeloid leukaemia (t-AML) or AML with myelodysplasia-related changes (AML-MRC). | PLGB 31626/0004/OD1 | 27-Aug-28 |
| Wainzua | EPLONTERSEN | Transthyretin-mediated amyloidosis | Authorised orphan indication: Wainzua is indicated for the treatment of hereditary transthyretin-mediated amyloidosis (ATTRv amyloidosis) in adult patients with Stage 1 and 2 polyneuropathy. | PLGB 17901/0377/OD1 | 14-Oct-34 |
| Waylivra | volanesorsen Phosphorothioate oligonucleotide targeted to apolipoprotein C-III | Familial chylomicronemia syndrome | Waylivra is indicated as an adjunct to diet in adult patients with genetically confirmed familial chylomicronemia syndrome (FCS) and at high risk for pancreatitis, in whom response to diet and triglyceride lowering therapy has been inadequate | PLGB 51704/0003/OD1 | 08-May-29 |
| Winrevair | Sotatercept | Pulmonary arterial hypertension | Winrevair, in combination with other pulmonary arterial hypertension (PAH) therapies, is indicated for the treatment of PAH in adult patients with WHO Functional Class (FC) II to III, to improve exercise capacity. | PLGB 53095/0104-107/OD1 | 27-Dec-34 |
| XENPOZYME | Olipudase alfa | Acid sphingomyelinase deficiency | Enzyme replacement therapy for the treatment of non-central nervous system (CNS) manifestations of acid sphingomyelinase deficiency in paediatric and adult patients with type A/B or type B. | PLGB 04425/0901/OD1 | 31-Jul-32 |
| Xermelo | telotristat (S)-ethyl 2-amino-3-(4-(2-amino-6-((R)-1-(4-chloro-2-(3-methyl-1H-pyrazol-1-yl)phenyl)-2,2,2-trifluoroethoxy)pyrimidin-4-yl)phenyl)propanoate | Carcinoid syndrome | Xermelo is indicated for the treatment of carcinoid syndrome diarrhoea in combination with somatostatin analogue (SSA) therapy in adults inadequately controlled by SSA therapy. | PLGB 28247/0008/OD1 | 20-Sep-27 |
| Yescarta | axicabtagene ciloleucel | Diffuse large B cell lymphoma | Treatment of adult patients with diffuse large B cell lymphoma (DLBCL) and high-grade B-cell lymphoma (HGBL) that relapses within 12 months from completion of, or is refractory to, first-line chemoimmunotherapy. Treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) after two or more lines of systemic therapy | PLGB 11972/0044/OD1 | 27-Aug-28 |
| Yescarta | axicabtagene ciloleucel | Primary mediastinal large B-cell lymphoma | YESCARTA is indicated for the treatment of adult patients with relapsed or refractory primary mediastinal large B-cell lymphoma (PMBCL) after two or more lines of systemic therapy. | PLGB 11972/0044/OD2 | 27-Aug-28 |
| Yescarta | axicabtagene ciloleucel | Follicular lymphoma | Treatment of adult patients with relapsed or refractory follicular lymphoma (FL) after three or more lines of systemic therapy’ | PLGB 11972/0044/OD3 | 09-Aug-32 |
| Yorvipath 168 micrograms/0.56 mL solution for injection in pre filled pen | PALOPEGTERIPARATIDE | Hypoparathyroidism | Treatment of hypoparathyroidism. | PLGB 51127/0001-0003/OD1 | 23/04/2034 |
| Yorvipath 294 micrograms/0.98 mL solution for injection in pre filled pen | PALOPEGTERIPARATIDE | Hypoparathyroidism | Treatment of hypoparathyroidism | PLGB 51127/0001-0003/OD1 | 23/04/2034 |
| Yorvipath 420 micrograms/1.4 mL solution for injection in pre filled pen | PALOPEGTERIPARATIDE | Hypoparathyroidism | Treatment of hypoparathyroidism. | PLGB 51127/0001-0003/OD1 | 23/04/2034 |
| Zejula | niraparib (3S)-3-{4-[7-(aminocarbonyl)-2H-indazol-2-yl] phenyl} piperidine tosylate monohydrate salt | Ovarian cancer | as monotherapy for the maintenance treatment of adult patients with advanced epithelial (FIGO Stages III and IV) high-grade ovarian, fallopian tube or primary peritoneal cancer who are in response (complete or partial) following completion of first-line platinum-based chemotherapy. as monotherapy for the maintenance treatment of adult patients with platinum sensitive relapsed high grade serous epithelial ovarian, fallopian tube, or primary peritoneal cancer who are in response (complete or partial) to platinum based chemotherapy | PLGB 19494/0294/OD1 | 20-Nov-27 |
| Zokinvy | lonafarnib | Hutchinson-Gilford progeria syndrome | Treatment of patients 12 months of age and older with a genetically confirmed diagnosis of Hutchinson-Gilford progeria syndrome or a processing-deficient progeroid laminopathy associated with either a heterozygous LMNA mutation with progerin-like protein accumulation or a homozygous or compound heterozygous ZMPSTE24 mutation. | PLGB 56709/0001-0002/ OD1 | 24-Aug-34 |
| Zolgensma | onasemnogene abeparvovec Adeno-associated viral vector serotype 9 containing the human SMN gene | Spinal muscular atrophy | patients with 5q spinal muscular atrophy (SMA) with a bi-allelic mutation in the SMN1 gene and a clinical diagnosis of SMA Type 1, or patients with 5q SMA with a bi-allelic mutation in the SMN1 gene and up to 3 copies of the SMN2 gene. | PLGB 53139/0002/OD1 | 18-May-30 |
| Ztalmy 50 mg/ml Oral suspension | GANAXOLONE | Cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder | indicated for the adjunctive treatment of epileptic seizures associated with cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder (CDD) in patients 2 to 17 years of age. Ztalmy may be continued in patients 18 years of age and older. | PLGB 55682/0001/OD1 | 07/03/2034 |