Reimbursement Approval of Evrysdi

Reimbursement Approval of Evrysdi

SMA Ireland Welcomes Reimbursement Approval of Evrysdi® for the Treatment of Spinal Muscular Atrophy

SMA Ireland is pleased to announce the significant step forward in the treatment landscape for spinal muscular atrophy (SMA) with the recent approval for reimbursement of Evrysdi® (risdiplam) in Ireland. This marks a significant milestone, making Evrysdi® the third SMA medicine accessible to patients in the country, alongside Spinraza and Zolgensma.

Developed by Roche Pharmaceutical, Evrysdi® stands out as a valuable addition to the portfolio of available SMA medicines, being the only oral treatment option for this debilitating condition.

SMA Ireland extends its heartfelt appreciation to the Medicines Management Program of the Health Service Executive (HSE) for their diligence and efforts in securing this approval. However, the organisation strongly expresses its concern that Evrysdi® has been approved only for patients under 18 years of age. With just approximately 70 individuals in Ireland affected by SMA, this decision potentially leaves 16 adults without access to this life-changing treatment.

While acknowledging the essential role of medical assessment in treatment decisions, SMA Ireland asserts that an arbitrary age cap contradicts the principles of fairness and equity. Unlike Zolgensma, which is limited to children up to approximately 24 months, and Spinraza, initially recommended only for under-18s due to limited data, Evrysdi® has clinical data supporting its efficacy for patients up to 60 years of age.

Notably, numerous European countries have approved Evrysdi® without age restrictions, including Belgium, Germany, Italy, Spain, and the United Kingdom, amongst others. SMA Ireland urges the Medicines Management Program to reconsider the age cap and assess the 16 currently excluded adults based on their medical needs.

In light of this momentous development, SMA Ireland calls for a fair and unbiased evaluation of the medical necessity and merits of providing Evrysdi® to adults as well. The organisation remains dedicated to supporting SMA patients and advocating for equitable access to life-changing treatments.

About Evrysdi® (risdiplam):

Risdiplam, marketed as Evrysdi®, received European Union authorisation in 2021 for the treatment of SMA Type 1, Type 2, or Type 3 patients with four or fewer copies of the SMN2 gene. Administered orally, Evrysdi® modifies SMN2 pre-mRNA splicing, enhancing the production of functional SMN protein. It is expected that the license for risdiplam will soon be extended to include patients less than two months of age.

For further details see: HSE Managed Access Protocols

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