Risdiplam / Evrysdi / RG7916
Pages in this website section last updated 18th March 2021
18th March 2021
Roche Presents New Two-Year Data From SUNFISH (Part 2) Trial
26th February 2021
CHMP Publishes Positive Opinion on Risdiplam
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 gives 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.
Once the treatment receives marketing authorisation the scheme starts to close. Marketing authorisation by the European Medicines Authority (EMA) affects Northern Ireland’s access to the scheme - this is now in process. Please read more detail about this, and the likely timeline to closure, here.
Marketing authorisation by the Medicines and Healthcare products Regulatory Agency (MHRA) affects England, Scotland and Wales. This application will happen soon. Please read more detail about this, and the likely timeline to closure, here.
The Patient Groups (PAGs) jointly collated questions about the scheme which Roche has answered - read more. Further questions raised by families and adults about clinical requirements are answered by clinicians and covered, here.
29 Trusts are now prescribing risdiplam via the EAMS. 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 26th February the European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) announced that it has recommended the approval of Evrysdi™ (risdiplam) for the treatment of 5q SMA in patients 2 months of age and older, with a clinical diagnosis of SMA Type 1, Type 2 or Type 3, or with one to four SMN2 copies.
The CHMP positive opinion is now referred to the European Commission (EC), which grants marketing authorisations for centrally authorised medicines in the European Union (EU). The EC will review the CHMP recommendation - a final decision regarding approval is expected from the European Commission in the next two months, but could be as early as the end of March.
As the UK left the European Union during the ongoing EMA assessment of risdiplam, Roche will submit risdiplam for marketing authorisation consideration by the MHRA (Medicines and Healthcare products Regulatory Agency). Risdiplam is one of the first medicines to undergo this transition approval pathway. As this is a new process it comes with some uncertainty as to the precise timelines, however Roche anticipate marketing authorisation for England, Scotland and Wales at some point in the next few months.
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 its licence / marketing authorisation (see above) and they may limit access based on their own assessment of the evidence for the clinical and cost effectiveness of a treatment.
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.
Roche has not yet made an application to the Scottish Medicines Consortium.
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.