Important Risdiplam Access Updates
21 May 2021
Updates yesterday were that:
- risdiplam now has GB marketing authorisation from the MHRA
- there’s a long way still to go before we know if NICE will recommend funding by the NHS
- good news that Roche will submit an application in June for NHS provision in Scotland
- the EAMS scheme for people with SMA Type 1 or 2 for whom nusinersen is not clinically suitable continues for those already enrolled
- the EAMS now closes to new people, unless they’ve been identified before the MHRA announcement. Anyone who thinks they would be eligible for the EAMS but who is not yet enrolled in the scheme should now contact their clinical team.
Here is the letter with all these updates that Roche sent to the patient groups today.
Dear Andi, Liz, Rob and Kate
In response to your request, I am pleased to be writing to inform you that risdiplam has on 20 May 2021 received marketing authorisation from the MHRA. This means risdiplam is now licensed in Great Britain and Northern Ireland "for the treatment of 5q spinal muscular atrophy (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." From this point risdiplam will have the brand name Evrysdi®.
What does this mean for the EAMS?
Following MHRA guidelines, the Early Access to Medicines Scheme (EAMS) for risdiplam is now closed in Great Britain for new patients (i.e. patients identified after marketing authorisation). However we have agreed with the MHRA that, for patients already identified at the time of Great Britain marketing authorisation, applications for these patients can be accepted after marketing authorisation up to and including 1 July 2021. These previously identified patients - should they fulfil the EAMS eligibility criteria - are therefore still able to receive risdiplam through the EAMS. Existing patients participating in the EAMS will continue to be treated under the terms of the EAMS.
Any questions from SMA patients and their families about the EAMS should be directed to a healthcare professional.
What happens next?
A National Institute for Health and Care Excellence (NICE) appraisal for risdiplam is currently underway. Until this appraisal is complete risdiplam will not be available to new patients on the NHS. The current NICE timeline for the appraisal anticipates publication of final guidance in July 2021. However, as in previous appraisals, this process could take longer.
Risdiplam will be submitted to the Scottish Medicines Consortium (SMC) in June 2021.
We are delighted to be working with you and the SMA community across the UK. If you have any questions about this latest milestone, please feel free to contact me.