Roche on Wednesday announced that its Evrysdi (risdiplam), indicated for the treatment of Spinal Muscular Atrophy (SMA), has shown positive outcomes. The results come from the new two-year-old data from the JEWELFISH study which is the largest ever conducted in previously treated SMA patients.
According to the company’s statement, the data showed Evrysdi improved or maintained motor function and led to rapid increases in SMN protein levels which were sustained after 2-years of treatment.
Moreover, the data will be presented at the 27th World Muscle Society (WMS) Congress, 11-15 October 2022.
“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. The findings add to our confidence when making treatment decisions for previously-treated patients in need,” said Dr. Claudia Chiriboga, Professor of Neurology and Paediatrics, Department of Neurology, Columbia University Medical Center, New York, USA in a statement.
The study enrolled 174 people out of which 36 percent were adults and 63 percent of participants had a very severe level of the disease. 83 perent had scoliosis, and 44 percent of the participants were earlier treated with nusinersen (Spinraza), 41 percent olesoxime and 8 percent with onasemnogene abeparvovec (Zolgensma).
SMA, a genetic disorder, leads to debilitating and potentially fatal muscle weakness. Studies suggest, it affects approximately one in 10,000 babies and is the leading genetic cause of infant mortality.
According to the study, the drug led to a two-fold increase in median SMN protein levels versus baseline after 4 weeks of treatment in all patient groups, irrespective of previous treatment.
“These important data demonstrate the safety and efficacy of Evrysdi in a broad, real-world population of people previously treated with an SMA-targeting therapy. Those enrolled in JEWELFISH had very severe disease, with over 80 percent having scoliosis, so maintaining motor function–especially for a progressive disease–can be potentially life-changing,” said Levi Garraway, M.D, Ph.D, Roche’s chief medical officer and head of global products in a statement.
In 2018, Evrysdi was granted PRIME designation by the European Medicines Agency (EMA) and Orphan Drug Designation by the US Food and Drug Administration in 2017. Last year, Evrysdi was awarded Drug Discovery of the Year by the British Pharmacological Society as well as the Society for Medicines Research award for Drug Discovery.
Currently, Evrysdi is currently approved in 91 countries and the dossier is under review in a further 18 countries.
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