Genentech Unveils Positive Two-Year Data For Spinal Muscular Atrophy Drug

Roche’s Genentech has revealed positive two-year data from the Jewelfish study on Evrysdi (risdiplam) in patients with Type 1, 2, or 3 spinal muscular atrophy (SMA) who have been treated with other SMA therapies, including Spinraza and Zolgensma. 

First approved in 2020 for patients aged between six months and 60 years, Evrysdi recently secured a label extension to include infants younger than two months old. 

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Treatments For Debilitating Muscle Weakness 

SMA is a severe and potentially fatal genetic disease that affects around one in 10,000 babies. It is caused by a mutation in the survival motor neuron 1 (SMN1) gene, which leads to a deficiency in SMN1 protein. Deficiency in SMN1 causes nerve cells to dysfunction, leading to progressive muscle weakness. 

Evrysdi, a survival motor neuron 2 (SMN2) splicing modifier, treats SMA by increasing the production of the SMN protein. 

According to the Jewelfish study, which Genentech has touted as having the “broadest and most diverse patient population” ever studied for SMA, Evrysdi treatment led to a two-fold increase in median SMN protein levels vs baseline after four weeks of treatment. The increased levels were sustained for over two years. 

Common side effects of Evrysdi in the trial include fever (24%), upper respiratory tract infection (21%), and headache (18%). Serious adverse events include pneumonia (3%) and respiratory failure (2%). The side effects were consistent with those observed in patients who never received treatment in the previous trials. Jewelfish also recorded low rates of discontinuation (5%) over the 24-month period. 

“The consistent safety profile and exploratory efficacy we have seen in the Jewelfish study, the largest ever conducted in previously treated patients, reinforces Evrysdi as a meaningful treatment option across SMA populations,” said Dr. Claudia Chiriboga, Professor of Neurology and Pediatrics, Department of Neurology, Columbia University Medical Center, New York. 

“The findings add to our confidence when making treatment decisions for previously-treated patients in need,” she said.

Biogen’s Spinraza and Novartis’ Zolgensma are approved treatments for SMA that come with hefty price tags. Evrysdi, coming in at around $23,000 according to GoodRx, is a fraction of its rivals’ prices. The latest trial data could potentially fire up the rivalry between Roche and its competitors. 

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Joy Lin

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