EMA approves third SMA treatment, Evrysdi (risdiplam)

EMA approves third SMA treatment, Evrysdi (risdiplam)

SMA Ireland are delighted that the European Commission has approved Evrysdi (risdiplam) for the treatment of 5q Spinal Muscular Atrophy in adults and children with SMA Types 1, 2 or 3.

Speaking of the approval, Karen Mannion, chair of SMA Ireland said, "Everysdi is taken orally for the treatment of Spinal Muscular Atrophy each day in the home. It has proven efficacy in patients up to 60 years old. SMA Ireland calls for the swift approval for reimbursement of Everysdi in Ireland.

SMA Ireland's top priority is that more reasonable onboarding criteria are devised with respect to Everysdi for SMA patients in Ireland. The arbitrary age cap applied to Spinraza was cruel and hard to justify. It is crucial that no such age restrictions are tagged to any reimbursement of Everysdi.

Adults with SMA have been excluded from any treatment in Ireland to date. Everysdi has extensive and positive drug trial data across a wide age range, including adults. This is a very positive day for SMA in Europe, and importantly offers adults with SMA in Ireland hope for the future. We must not let them down!"

Background:

There are now three drugs targeting SMA: Spinraza (Biogen), Zolgensma (Novartis) and Everysdi (Roche).

Only one of these drugs is currently available in Ireland.

- Spinraza was approved for patients under 18 years of age for reimbursement in Ireland in 2019.
- SMA Ireland expects Zolgensma to receive approval for infants from the HSE later this year.
- Everysdi maker, Roche, may now apply to the HSE for reimbursement of Everysdi in Ireland.

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