Decision
Expired orphan registrations
Updated 3 November 2025
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| Product name | Active substance | Orphan Condition | OD Indication | Designation number | Orphan Market Exclusivity Expiry date | Date Orphan designation removed (if different to expiry) |
|---|---|---|---|---|---|---|
| ADAKVEO | crizanlizumab | Sickle cell disease | Adakveo is indicated for the prevention of recurrent vaso-occlusive crises (VOCs) in sickle cell disease patients aged 16 years and older. It can be given as an add on therapy to hydroxyurea/hydroxycarbamide (HU/HC) or as monotherapy in patients for whom HU/HC is inappropriate or inadequate. | PLGB 00101/1191/OD1 | 29 October 2030 | 10-Jan-24 |
| ADCETRIS | brentuximab vedotin, Monoclonal antibody against human CD30 covalently linked to the cytotoxin monomethylauristatin E | Systemic anaplastic large cell lymphoma. | ADCETRIS in combination with cyclophosphamide, doxorubicin and prednisone (CHP) is indicated for adult patients with previously untreated systemic anaplastic large cell lymphoma (sALCL). ADCETRIS is indicated for the treatment of adult patients with relapsed or refractory sALCL. | PLGB 16189/0093/OD1 | 30 October 2024 | Not set |
| ADCETRIS | brentuximab vedotin, Monoclonal antibody against human CD30 covalently linked to the cytotoxin monomethylauristatin E | Hodgkin lymphoma | ADCETRIS is indicated for adult patients with previously untreated CD30+ Stage IV Hodgkin lymphoma (HL) in combination with doxorubicin, vinblastine and dacarbazine (AVD). ADCETRIS is indicated for the treatment of adult patients with CD30+ HL at increased risk of relapse or progression following autologous stem cell transplant (ASCT). ADCETRIS is indicated for the treatment of adult patients with relapsed or refractory CD30+ Hodgkin lymphoma (HL): 1. following ASCT, or 2. following at least two prior therapies when ASCT or multi-agent chemotherapy is not a treatment option. | PLGB 16189/0093/OD2 | 30 October 2024 | Not set |
| Adempas | riociguat, Methyl 4,6-diamino-2-[1-(2-fluorobenzyl)-1H-pyrazolo[3,4-b]pyridine-3-yl]-5-pyrimidinyl(methyl)carbamate | Chronic thromboembolic pulmonary hypertension (CTEPH) | Adempas is indicated for the treatment of adult patients with WHO Functional Class (FC) II to III with - inoperable CTEPH - persistent or recurrent CTEPH after surgical treatment, to improve exercise capacity. Pulmonary arterial hypertension (PAH) Adempas, as monotherapy or in combination with endothelin receptor antagonists, is indicated for the treatment of adult patients with pulmonary arterial hypertension (PAH) with WHO Functional Class (FC) II to III to improve exercise capacity. Efficacy has been shown in a PAH population including aetiologies of idiopathic or heritable PAH or PAH associated with connective tissue disease. | PLGB 00010/0670 – 0674/OD1 | 31 March 2024 | Not set |
| AMVUTTRA | Vutrisiran Sodium | Transthyretin-mediated amyloidosis | Treatment of hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) in adult patients with stage 1 or stage 2 polyneuropathy. | PLGB 50597/0006/OD1 | 16 September 2032 | Apr-25 |
| Bronchitol | Mannitolum | Cystic fibrosis | Bronchitol is indicated for the treatment of cystic fibrosis (CF) in adults aged 18 years and above as an add-on therapy to best standard of care. | PLGB 27944/0002/OD1 | 18 April 2022 | Not set |
| Carbaglu | Carglumic acid | Isovaleric acidaemia | Carbaglu is indicated in treatment of hyperammonaemia due to isovaleric acidaemia. | PLGB 15266/0019/OD1 | 01 June 2021 | Not set |
| Carbaglu | Carglumic acid | Methylmalonic acidaemia | Carbaglu is indicated in treatment of hyperammonaemia due to methymalonic acidaemia. | PLGB 15266/0019/OD2 | 01 June 2021 | Not set |
| Carbaglu | Carglumic acid | Propionic acidaemia | Carbaglu is indicated in treatment of hyperammonaemia due to propionic acidaemia. | PLGB 15266/0019/OD3 | 01 June 2021 | Not set |
| Cerdelga | eliglustat | Gaucher Disease (1R, 2R)-Octanoic acid [2-(2’,3’-dihydro-benzo [1,4] dioxin-6’-yl)-2-hydroxy-1-pyrrolidin-1-ylmethyl-ethyl]-amide-L-tartaric acid salt | 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 January 2025 | Not set |
| Columvi 2.5 mg and 10mg concentrate for solution for infusion | GLOFITAMAB | Diffuse large B-cell lymphoma | Treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma (R/R DLBCL), after two or more lines of systemic therapy. | PLGB 00031/0931-0932/OD1 | 16 October 2033 | Apr-25 |
| Cometriq | cabozantinib, Cyclopropane-1,1-dicarboxylic acid [4-(6,7-dimethoxy-quinolin-4-yloxy)-phenyl]-amide (4-fluoro-phenyl)-amide, (L)-malate salt | Medullary thyroid carcinoma | COMETRIQ is indicated for the treatment of adult patients with progressive, unresectable locally advanced or metastatic medullary thyroid carcinoma. For patients in whom rearranged during transfection (RET) mutation status is not known or is negative, a possible lower benefit should be taken into account before individual treatment decision. | PLGB 28247/0004 – 0005/OD1 | 26 March 2024 | Not set |
| Dacogen | decitabine | Acute myeloid leukaemia | Dacogen is indicated for the treatment of adult patients with newly diagnosed de novo or secondary acute myeloid leukaemia (AML), according to the World Health Organisation (WHO) classification, who are not candidates for standard induction chemotherapy. | PLGB 00242/0662/OD1 | 24 September 2024 | Not set |
| Defitelio | defibrotide | Hepatic veno-occlusive disease | Treatment of severe hepatic veno-occlusive disease (VOD) also known as sinusoidal obstructive syndrome (SOS) in haematopoietic stem-cell transplantation (HSCT) therapy. It is indicated in adults and in adolescents, children and infants over 1 month of age. | PLGB 31626/0005/OD1 | 22 October 2023 | Not set |
| Esbriet | pirfenidone | Idiopathic pulmonary fibrosis | Esbriet is indicated in adults for the treatment of mild to moderate Idiopathic Pulmonary Fibrosis (IPF). | PLGB 00031/0851 – 0854/OD1 | 02 March 2021 | Not set |
| Evrysdi | risdiplam | Spinal Muscular Atrophy | Evrysdi is indicated for the treatment of 5q spinal muscular atrophy (SMA) in patients 2 months of age and older, with a clinical diagnosis of SMA Type 1, Type 2 or Type 3 or with one to four SMN2 copies. | PLGB 00031/0920/OD1 | 19 May 2031 | Dec-23 |
| Farydak | panobinostat | Treatment of multiple myeloma | Farydak, in combination with bortezomib and dexamethasone, is indicated for the treatment of adult patients with relapsed and/or refractory multiple myeloma who have received at least two prior regimens including bortezomib and an immunomodulatory agent. | PLGB 54599/0008-0010/OD1 | 01 September 2025 | Not set |
| Gazyvaro | obinutuzumab | Chronic lymphocytic leukaemia (CLL) | Gazyvaro in combination with chlorambucil is indicated for the treatment of adult patients with previously untreated CLL and with comorbidities making them unsuitable for full-dose fludarabine based therapy (see section 5.1). | PLGB 00031/0856/OD1 | 24 July 2024 | Not set |
| Granupas | Para-aminosalicylic acid | Tuberculosis | Indicated for use as part of an appropriate combination regimen for multi-drug resistant tuberculosis in adults and paediatric patients from 28 days of age and older when an effective treatment regimen cannot otherwise be composed for reasons of resistance or tolerability. Consideration should be given to official guidance on the appropriate use of antibacterial agents. | PLGB 35068/0003/OD1 | 09 April 2024 | Not set |
| Hepcludex | bulevirtide | Hepatitis delta virus infection | The treatment of chronic hepatitis delta virus (HDV) infection in plasma (or serum) HDV-RNA positive adult patients with compensated liver disease. | PLGB 50662/0002/OD1 | 05 August 2030 | Sep-21 |
| Hetlioz | tasimelteon | Non-24-hour sleep-wake disorders in blind people with no light perception | HETLIOZ is indicated for the treatment of Non-24-Hour Sleep-Wake Disorder (Non-24) in totally blind adults. | PLGB 54231/0001/OD1 | 07 July 2025 | Not set |
| Iclusig | ponatinib Benzamide, 3-(2-imidazo[1,2-b]pyridazin-3-ylethynyl)-4-methyl-N-[4-[(4-methyl-1-piperazinyl)methyl]-3-(trifluoromethyl)phenyl] | Acute lymphoblastic leukaemia | Iclusig is indicated in adult patients with Philadelphia chromosome positive acute lymphoblastic leukaemia (Ph+ ALL) who are resistant to dasatinib; who are intolerant to dasatinib and for whom subsequent treatment with imatinib is not clinically appropriate; or who have the T315I mutation. | PLGB 49635/0001 – 0003/OD1 | 03 July 2023 | Not set |
| Iclusig | ponatinib Benzamide, 3-(2-imidazo[1,2-b]pyridazin-3-ylethynyl)-4-methyl-N-[4-[(4-methyl-1-piperazinyl)methyl]-3-(trifluoromethyl)phenyl] | Chronic myeloid leukaemia | Iclusig is indicated in adult patients with chronic phase, accelerated phase, or blast phase chronic myeloid leukaemia (CML) who are resistant to dasatinib or nilotinib; who are intolerant to dasatinib or nilotinib and for whom subsequent treatment with imatinib is not clinically appropriate; or who have the T315I mutation | PLGB 49635/0001 – 0003/OD2 | 03 July 2023 | Not set |
| Imnovid | pomalidomide | Multiple myeloma | Imnovid in combination with dexamethasone is indicated in the treatment of adult patients with relapsed and refractory multiple myeloma who have received at least two prior treatment regimens, including both lenalidomide and bortezomib, and have demonstrated disease progression on the last therapy | PLGB 50412/0002 – 0005/OD1 | 08 August 2023 | Not set |
| Kalydeco | ivacaftor N-(2,4-Di-tert-butyl-5-hydroxyphenyl)-1,4-dihydro-4-oxoquinoline-3-carboxamide | Cystic fibrosis | Treatment of cystic fibrosis (CF) in patients age six years and older who have a G551D mutation in the CFTR gene | PLGB 22352/0008 – 0011/OD1 | 25 July 2022 | Not set |
| Kalydeco | ivacaftor N-(2,4-Di-tert-butyl-5-hydroxyphenyl)-1,4-dihydro-4-oxoquinoline-3-carboxamide | Cystic fibrosis | In a combination regimen with ivacaftor /tezacaftor /elexacaftor tablets for the treatment of adults, adolescents and children aged 6 years and older with cystic fibrosis (CF) who have at least one F508del mutation in the CFTR gene (see section 5.1) | PLGB 22352/0008/OD1, PLGB 22352/0013/OD1 | 25 July 2022 | Not set |
| Kanuma | sebelipase alfa Recombinant human lysosomal acid lipase | Lysosomal acid lipase deficiency | Kanuma is indicated for long-term enzyme replacement therapy (ERT) in patients of all ages with lysosomal acid lipase (LAL) Deficiency. | PLGB 31187/0017/OD1 | 01 September 2025 | Not set |
| Ketoconazole HRA | Ketoconazole | Cushing’s syndrome | Ketoconazole HRA is indicated for the treatment of endogenous Cushing’s syndrome in adults and adolescents above the age of 12 years. | PLGB 51757/0002/OD1 | 21 November 2024 | Not set |
| Mozobil | plerixafor l, 1’-[1,4-phenylenebis (methylene)]-bis-1,4,8,11- tetraazacyclotetradecane | Mobilization of progenitor cells prior to stem cell transplantation | Adult patients Mozobil is indicated in combination with granulocyte-colony stimulating factor (G-CSF) to enhance mobilisation of haematopoietic stem cells to the peripheral blood for collection and subsequent autologous transplantation in adult patients with lymphoma or multiple myeloma whose cells mobilise poorly. Paediatric patients (1 to less than 18 years) Mozobil is indicated in combination with G-CSF to enhance mobilisation of haematopoietic stem cells to the peripheral blood for collection and subsequent autologous transplantation in children with lymphoma or solid malignant tumours, either: - pre-emptively, when circulating stem cell count on the predicted day of collection after adequate mobilization with G-CSF (with or without chemotherapy) is expected to be insufficient with regards to desired hematopoietic stem cells yield, or - who previously failed to collect sufficient haematopoietic stem cells. | PLGB 04425/0769/OD1 | 04 August 2021 | Not set |
| Nexavar | sorafenib tosylate | Follicular thyroid cancer | Nexavar is indicated for the treatment of patients with progressive, locally advanced or metastatic, differentiated (follicular/Hürthle cell) thyroid carcinoma, refractory to radioactive iodine. | PLGB 00010/0701/OD1 | 27 May 2024 | Not set |
| Nexavar | sorafenib tosylate | Papillary thyroid cancer | Nexavar is indicated for the treatment of patients with progressive, locally advanced or metastatic, differentiated (papillary) thyroid carcinoma, refractory to radioactive iodine. | PLGB 00010/0701/OD2 | 27 May 2024 | Not set |
| NexoBrid | Concentrate of proteolytic enzymes enriched in bromelain Purified bromelain | Partial deep dermal and full thickness burns | NexoBrid is indicated for removal of eschar in adults with deep partial- and full-thickness thermal burns. | PLGB 43948/0002 – 0003/OD1 | 20 December 2022 | Not set |
| Opsumit | macitentan | Pulmonary arterial hypertension | Opsumit, as monotherapy or in combination, is indicated for the long-term treatment of pulmonary arterial hypertension (PAH) in adult patients of WHO Functional Class (FC) II to III. Efficacy has been shown in a PAH population including idiopathic and heritable PAH, PAH associated with connective tissue disorders, and PAH associated with corrected simple congenital heart disease. | PLGB 00242/0663/OD1 | 27 December 2023 | Not set |
| Plenadren | hydrocortisone | Adrenal insufficiency | Treatment of adrenal insufficiency in adults. | PLGB 39534/0009-0010/OD1 | 14 November 2021 | Not set |
| Raxone | idebenone | Leber’s hereditary optic neuropathy | Raxone is indicated for the treatment of visual impairment in adolescent and adult patients with Leber’s Hereditary Optic Neuropathy (LHON). | PLGB 08829/0204/OD1 | 10 September 2025 | Not set |
| Revestive | teduglutide [gly2] Recombinant human glucagon-like peptide | Short Bowel Syndrome | Revestive is indicated for the treatment of patients aged 1 year and above with Short Bowel Syndrome. Patients should be stable following a period of intestinal adaptation after surgery. | PLGB 16189/0077 – 0078/OD1 | 04 September 2024 | Not set |
| SCENESSE | afamelanotide [Nle4, D-Phe7]-alpha-melanocyte stimulating hormone | Erythropoietic protoporphyria | Scenesse is indicated for prevention of phototoxicity in adult patients with erythropoietic protoporphyria (EPP). | PLGB 30619/0002/OD1 | 29 December 2024 | Not set |
| Signifor | pasireotide | Acromegaly | Treatment of adult patients with acromegaly for whom surgery is not an option or has not been curative and who are inadequately controlled on treatment with another somatostatin analogue. The 60 mg strength is only to be used in the treatment of acromegaly. | PLGB 15266/0032 – 0039/OD1 | 21 November 2024 | Not set |
| Signifor | pasireotide | Cushing’s disease | Treatment of adult patients with Cushing’s disease for whom surgery is not an option or for whom surgery has failed. | PLGB 15266/0032 – 0039/OD2 | 27 April 2022 | Not set |
| Sirturo 20 mg Tablets | BEDAQUILINE FUMARATE | tuberculosis | as per section 4.1 of the SPC | PLGB 00242/0744 /OD1 | 07 March 2024 | Not set |
| Soliris | eculizumab | Atypical haemolytic uremic syndrome | Soliris is indicated in adults and children for the treatment of atypical haemolytic uremic syndrome (aHUS). | PLGB 31775/0003/OD1 | 29 November 2023 | Not set |
| Strensiq | asfotase alfa Recombinant human tissue non-specific alkaline phosphatase - Fc - deca-aspartate fusion protein | Hypophosphatasia | Strensiq is indicated for long-term enzyme replacement therapy in patients with paediatric-onset hypophosphatasia to treat the bone manifestations of the disease. | PLGB 31775/0004 – 0005/OD1 | 01 September 2025 | Not set |
| Sylvant | siltuximab Chimeric-anti-interleukin-6 monoclonal antibody | Castleman’s disease | Sylvant is indicated for the treatment of adult patients with multicentric Castleman’s disease (MCD) who are human immunodeficiency virus (HIV) negative and human herpesvirus 8 (HHV 8) negative. | PLGB 44185/0006 – 0007/OD1 | 27 May 2024 | Not set |
| TOBI Podhaler | tobramycin | Pseudomonas aeruginosa lung infection in cystic fibrosis | TOBI Podhaler is indicated for the suppressive therapy of chronic pulmonary infection due to Pseudomonas aeruginosa in adults and children aged 6 years and older with cystic fibrosis. Consideration should be given to official guidance on the appropriate use of antibacterial agents. | PLGB 46302/0222/OD1 | 25 July 2023 | Not set |
| Translarna | ataluren (3-[5-(2-fluoro-phenyl)-[1,2,4]oxadiazole-3-yl]-benzoic acid | Duchenne muscular dystrophy | Translarna is indicated for the treatment of Duchenne muscular dystrophy resulting from a nonsense mutation in the dystrophin gene, in ambulatory patients aged 2 years and older. The presence of a nonsense mutation in the dystrophin gene should be determined by genetic testing | PLGB 44221/0002 – 0004/OD1 | 05 August 2024 | Not set |
| Vimizim | elosulfase alfa Recombinant human N-acetylgalactosamine-6-sulfatase | Mucopolysaccharidosis, type IVA (Morquio A Syndrome) | Vimizim is indicated for the treatment of mucopolysaccharidosis, type IVA (Morquio A Syndrome, MPS IVA) in patients of all ages. | PLGB 45814/0007/OD1 | 30 April 2024 | Not set |
| VPRIV | velaglucerase alfa | Gaucher disease | VPRIV is indicated for long-term enzyme replacement therapy (ERT) in patients with type 1 Gaucher disease. | PLGB 16189/0088/OD1 | 30 August 2022 | Not set |
| Vyndaqel | tafamidis N-methyl D-(2,3,4,5,6-pentahydroxy-hexyl)-ammonium; 2-(3,5-dichloro-phenyl)-benzoxazole-6-carboxylate | Familial amyloid polyneuropathy | Vyndaqel is indicated for the treatment of transthyretin amyloidosis in adult patients with stage 1 symptomatic polyneuropathy to delay peripheral neurologic impairment. | PLGB 00057/1660/OD1 | 18 November 2021 | Not set |
| Welireg | BELZUTIFAN | von Hippel-Lindau (VHL) disease | Treatment of adult patients with von Hippel-Lindau (VHL) disease who require therapy for VHL-associated renal cell carcinoma (RCC), central nervous system (CNS) hemangioblastomas, or pancreatic neuroendocrine tumours (pNET), and for whom localised procedures are unsuitable or undesirable. | PLGB 53095/0087/OD1 | 30 May 2032 | Nov-23 |
| Xaluprine | mercaptopurine Mercaptopurine (oral suspension) | Acute lymphoblastic leukaemia | Treatment of acute lymphoblastic leukaemia (ALL) in adults, adolescents and children. | PLGB 13581/0002/OD1 | 13 March 2022 | Not set |