Decision

Early access to medicines scheme (EAMS) scientific opinion: Raxone to treat the decline of respiratory function in patients with Duchenne Muscular Dystrophy

Renewal of EAMS scientific opinion issued to Santhera Pharmaceuticals for Raxone to treat the decline of respiratory function in patients with Duchenne Muscular Dystrophy.

Documents

Raxone - Public assessment report (PAR)

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Annex to PAR Raxone EAMS Renewal

Treatment protocol for patients

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Treatment protocol for healthcare professionals

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Treatment protocol on the pharmacovigilance system

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Raxone - background information for medical directors

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Details

The renewed scientific opinion was issued on 21 June 2018 to Santhera Pharmaceuticals for Raxone to treat the decline of respiratory function in patients with Duchenne Muscular Dystrophy (DMD). The opinion is for patients from the age of 10 years who are currently not taking glucocorticoids.

The renewed scientific opinion includes:

  • a public assessment report including annex (updated 21 June 2018)

as well as treatment protocols:

  • for healthcare professionals (updated 21 June 2018)
  • for patients (updated 21 June 2018)
  • on the pharmacovigilance system (updated 21 June 2018)
  • EAMs scientific opinion – background information for Medical Directors

Contact

Information and details regarding patient access

For new patients wishing to access EAMS medicines in England, trusts must register patients on the NHS England web based registration system. Queries to NHS England regarding the scheme can be submitted to england.eams@nhs.net.

For information about access in Northern Ireland, contact Mark Timoney, Chief Pharmaceutical Officer at Mark.Timoney@dhsspsni.gsi.gov.uk and Joe Magee, Secondary Care Directorate at Joe.Magee@dhsspsni.gov.uk.

For information about access in Scotland contact the Scottish Government Directorate General Health & Social Care at John.Hannah@gov.scot and Rosemarie.parr@gov.scot

For information about access in Wales, contact the Welsh Government Department of Health and Social Services by emailing Andrew Evans, Interim Chief Pharmaceutical Officer at Andrew.Evans@wales.gsi.gov.uk and Karen Eveleigh, Head of Pharmacy and Prescribing policy at Karen.Eveleigh@wales.gsi.gov.uk.

Published 21 June 2017
Last updated 21 June 2018 + show all updates
  1. Issue of EAMS renewal, including publication of four new documents, replacing documents previously hosted on the page.
  2. Change of text under Contact
  3. First published.