We were all delighted to learn that the European Medicines Agency (EMA) approved Evrysdi (Risdiplam), at the end of March. Roche will now begin the process of applying to the HSE for reimbursement of the drug here.
Similar to Spinraza, Evrysdi is an "SMN2 exon 7 splice modifier". However, Evrysdi may be administered orally and is therefore an exciting alternative for those unable to tolerate the Spinraza spinal injection. Importantly, clinical trials for Evrysdi included very positive results for adults up to 60 years of age.
Roche set up a Compassionate Use Programme for early access to Evrysdi. This was handled by patients' doctors, who applied directly to Roche. On 31 March 2021, SMA Ireland received notice from Roche that following EMA approval of their SMA drug, Evrysdi, the Compassionate Use Programme is now formally closed for new patients. Roche confirmed that a total of 9 Irish patients were enrolled in the program, with no outstanding patient requests. These patients comprised both children and adults and form an important sample group for Evrysdi in Ireland. They will receive Evrysdi for a defined period, courtesy of Roche. Longer term treatment depends on the drug being approved for reimbursement by the HSE.
If you are one of the Evrysdi Compassionate Use Programme recipients and you would like to share your story with the SMA Ireland community, we would love to hear from you! You are the tip of the spear in our fight for this drug to be approved for reimbursement in Ireland!
SMA Ireland strongly believes that Evrysdi must to be added to the list of available treatments for people with SMA in Ireland. Unlike Spinraza, which is restricted in Ireland to people under 18 years of age, Evrysdi has valid trial data for both children and adults. It is imperative that the Evrysdi onboarding process does not include a similar arbitrary age cap. The fact that we have a small number of adults already receiving the drug via the Compassionate Use Programme gives us all hope that we can prove the efficacy of treatment for all age groups here.
These are very exciting times for Spinal Muscular Atrophy, but it is more important than ever that we all stick together and demand that Irish sufferers are given access to the full range of treatments now available.
Very best wishes,
Chair, SMA Ireland