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, including the public assessment report.

• From: Medicines and Healthcare products Regulatory Agency
• Published: 21 June 2017
• Last updated: 26 October 2020 — See all updates

Documents

Raxone - Public assessment report (PAR)

PDF, 771 KB, 4 pages

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Annex to PAR Raxone EAMS Renewal

PDF, 704 KB, 3 pages

Annex to PAR Raxone EAMS Second Renewal

PDF, 710 KB, 2 pages

Annex to PAR Raxone EAMS Third Renewal

PDF, 716 KB, 2 pages

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Treatment protocol for patients

PDF, 192 KB, 5 pages

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Treatment protocol for healthcare professionals

PDF, 292 KB, 11 pages

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Treatment protocol on the pharmacovigilance system

PDF, 145 KB, 4 pages

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Raxone - background information for medical directors

PDF, 80.3 KB, 1 page

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Details

The renewed scientific opinion was issued on the 21 June 2019 to Santhera Pharmaceuticals for Raxone to treat the decline of respiratory function in patients with Duchenne Muscular Dystrophy (DMD) 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 2019)

• a treatment protocol:

  • for healthcare professionals (updated 21 June 2019)
  • for patients (updated 21 June 2019)
  • on the pharmacovigilance system (updated 21 June 2019)
  • 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 Chief Pharmaceutical Officer 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 medicines.policy@gov.scot.

For information about access in Wales, contact the Welsh Government Health and Social Services Group by emailing Andrew Evans, Chief Pharmaceutical Officer or Lynne Schofield, Head of Pharmacy and Prescribing policy at pharmacyand.prescribingbranch@gov.wales.

Published 21 June 2017
Last updated 26 October 2020 + show all updates

1. 26 October 2020

   Removal of scientific opinion for Raxone due to discontinuation of EAMS programme at the request of the company

2. 22 June 2020

   The Annex to Public Assessment Report THIRD RENEWAL was added

3. 30 December 2019

   Changes of multiple contacts in Contacts section.

4. 24 June 2019

   Updated contact details information and details regarding patient access

5. 21 June 2019

   Updated documents and content for 2019 and added a second annexe to PAR Raxone EAMS Renewal document

6. 21 June 2018

   Issue of EAMS renewal, including publication of four new documents, replacing documents previously hosted on the page.

7. 4 June 2018

   Change of text under Contact

8. 21 June 2017

   First published.