Risdiplam in the treatment of type 1 and type 2 spinal muscular atrophy (SMA) in patients 2 months of age and older
EAMS scientific opinion issued to Roche Products Limited for Risdiplam 0.75 mg/ml powder for oral solution in the treatment of type 1 and type 2 Spinal Muscular Atrophy (SMA) in patients 2 months and older who are not suitable for authorised treatments.
Documents
Details
For the full EAMS indication please see section 4.1 of the Treatment protocol: Information for healthcare professionals.
The scientific opinion includes:
- public assessment report (PAR)
- a treatment protocol:
- for healthcare professionals
- for patients
- on the pharmacovigilance system
- information for NHS Medical Directors
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.