Risdiplam / Evrysdi / RG7916
Pages in this website section last updated 2nd October 2020
2nd October 2020
Roche: Update on Part 1 of the FIREFISH Risdiplam Clinical Trial
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.
The Patient Groups (PAGs) jointly collated questions about the scheme which Roche have provided answers to - read more.
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. The first 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.
We are now working with MDUK to get our joint patient groups submission to NICE by the deadline of 30th October 2020. We want it to be based on the views of the Community, so if you haven’t already responded to our survey about this please do so by 11th October. You can find the survey here. You can read our 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.