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Genentech's Evrysdi becomes first FDA-approved oral treatment for spinal muscular atrophy

Published on 10/08/20 at 11:08am

Genentech’s Evrysdi (risdiplam) has secured FDA approval for the treatment of spinal muscular atrophy (SMA), making it the first oral therapy available for the rare hereditary genetic condition in US and the third overall to be approved for the disease.

Data supporting the decision, made under Fast Track and Priority Review designation, were drawn from two clinical trials of patients with infantile-onset and later-onset SMA.

The first study in infantile-onset SMA evaluated the drug in 21 infant patients with an average age of 6.7 months at launch. Data from the study revealed that 41% were able to sit without support for at least five seconds, compared to natural progression in the disease where almost infants with the condition cannot site independently.

Furthermore, 81% of participants receiving Evrysdi survived for at least 23 months without permanent ventilation.

In the second study of later-onset SMA evaluating 180 patients between the ages of two and 25, use of Evrysdi generated an average increase from baseline of 1.36 on the motor function test MFM32 one year after initial treatment, compared to an increase of just 0.19 with placebo.

 “This approval comes at a time when access to treatment is more critical than ever as we navigate the COVID-19 pandemic,” remarked Dr Levi Garraway, Chief Medical Officer and Head of Global Product Development at Roche, Genentech’s parent company. “Evrysdi, with its at-home delivery made possible by oral dosing, represents an important and timely advancement for people with SMA. During these challenging times, we are extremely proud to bring this ground-breaking medicine to those living with this highly disabling, rare neurological condition.”

Matt Fellows

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