The purpose of SMA Europe is to provide a framework to stimulate collaboration and accelerate translational research pathways in SMA and promote patient care.
SMA Europe is pleased to share a community letter from Roche announcing a pre-approval access/ compassionate use programme for its investigational drug risdiplam. This will initially be for Type 1 SMA but will expand to Type 2 SMA upon filing for market authorisation. Dear Members of SMA Europe, Following your recent request to receive more […]
The United States’ Food and Drug Administration (FDA) has granted priority review for the New Drug Application (NDA) for risdiplam (RG7916) for the treatment of SMA. The FDA’s decision will be announced on 24th May 2020. If approved, risdiplam, an orally administered liquid, would be the first at-home administered medicine for people living with SMA. […]
Roche has announced positive data from the pivotal Part 2 of SUNFISH, a study evaluating risdiplam in people aged 2-25 years with Type 2 or 3 SMA: The study met its primary endpoint of change from baseline in a motor function test (the Motor Function Measure 32 – MFM-32 scale) after one year of treatment […]
Novartis announced this morning that the Food and Drug Administration (FDA) in the US has placed a partial hold on AVXS-101 intrathecal clinical trials for SMA patients based on findings in a small pre-clinical animal study. Zolgensma® , also known as AVXS-101 intravenous administration is not impacted and remains available in the US. Novartis is […]
Biogen has announced that it will conduct a new global clinical trial, DEVOTE. The study will evaluate if higher dosing of nusinersen can provide improved efficacy in the treatment of SMA across a broad population of patients. The study aims to further build on the long-term safety profile and efficacy of nusinersen in a projected […]