Risdiplam / Evrysdi / RG7916 

Pages in this website section last updated 23rd Dec 2020

Recent Headlines

23rd December 2020
Cambridge University Hospitals NHS Foundation Trust Joins the Risdiplam EAMS for Adults

21st December 2020
Roche - End of Year Summary

4th December 2020
Action to Address Lack of Access to the Risdiplam Early Access to Medicines Scheme for Adults in the East of England

You can find our Scientific Correspondent’s September 2020 summary of information about Risdiplam, how it works and the clinical trial results, here, and a summary table of the outcomes of the four trials: FIREFISH, SUNFISH, JEWELFISH and RAINBOWFISH, here.

Scroll down further for:

  • Early Access to Medicines Scheme
  • Progress Towards Licensing and Access
  • Global Pre-Approval Access Programme

Early Access to Medicines Scheme

On 17th September 2020, the Medicines and Healthcare Products Regulatory Agency (MRHA) announced an EAMS that will give access to Risdiplam for those who have SMA Type 1 or Type 2, aged 2 months and older, who are not suitable for authorised treatments.

You can find the announcement on the MRHA website 
here, and their patient information leaflet for anyone enrolled in the scheme, here.

The Patient Groups (PAGs) jointly collated questions about the scheme which Roche have provided answers to - read more.

More than 15 Trusts are now prescribing risdiplam via the Early Access to Medicines Scheme. Jointly with MDUK and the Community, we continue to support the further roll out of the EAMS across the UK, working with local neuromuscular 
teams and Trusts wherever there are challenges.

Progress Towards Licensing and Access

Roche is seeking a broad label for the treatment of SMA in paediatric and adult patients – that’s access for those who have SMA Type 1, Type 2 and Type 3.


On 17th August 2020 the European Medicines Agency (EMA) confirmed that it had accepted Roche’s submission requesting a licence to market risdiplam in Europe and would begin its formal scientific evaluation process of the drug. This is done by EMA’s Committee for Medicinal Products for Human Use (CHMP). The EMA has agreed to accelerate this assessment, which means that the evaluation process can be reduced from 15 to 9 months, if the accelerated assessment timetable is maintained. This could mean a decision by mid May 2021.


The National Institute for Health and Care Excellence (NICE) and the Scottish Medicines Consortium (SMC) are the two regulatory authorities that manage access to licensed medicines in the UK. Currently, they may not recommend access wider than an EMA licence (see above) and they may limit access based on their own assessment of the evidence for the clinical and cost effectiveness of a treatment. Roche is working with both authorities.

NICE has started its appraisal process. NICE’s website page on the risdiplam appraisal currently states that it will publish a decision about access on 21st July 2021. Thank you to those of you who completed the survey we ran jointly with MDUK about your views on whether NICE should recommend that NHS England funds risdiplam. Your responses were crucial for the submission we made together to NICE on 30th October 2020. You can view what we said and survey results, here.

The first NICE committee meeting is scheduled for 11th May 2021. As an Early Access to Medicines Scheme (EAMS) is already in place, any recommendation by NICE that the treatment should be provided the NHS should be enacted within 30 days.

You can read our earlier February 2020 comments in response to NICE’s pre-referral ‘draft scoping’ response request here




In November 2019, the Food and Drug Administration (FDA) granted risdiplam priority review as a New Drug Application (NDA). On 7th August 2020, the FDA approved risdiplam for the treatment of 5q SMA in adults and children 2 months of age and older.

Global Pre-Approval Access Programme

Roche’s Pre-Approval Access (PAA) / Compassionate Use Programme (CUP) for risdiplam was available in the UK from 17th August – 17th September 2020. A CUP enables patients who are facing the most urgent medical need and have no other treatment options, to access investigational therapies before these receive regulatory approval.

Access is strictly regulated and varies between countries. FAQs about how it worked are here.