The U.S. Food and Drug Administration approved the first oral drug to treat patients two months of age and older with spinal muscular atrophy (SMA) that can be taken at home.
Roche subsidiary Genentech’s Evrysdi (risdiplam) is the third FDA-approved drug to treat this rare and often fatal genetic disease affecting muscle strength and movement.
SMA is a hereditary disease that causes weakness and muscle wasting because patients lose lower motor neurons (nerve cells) that control skeletal movement muscles. It is a genetic disorder that often affects babies and children and makes it hard for them to use their muscles.
The approval was given based on the results of two clinical trials, where Evrysdi improved motor function in patients at various levels of disease severity, including Types 1, 2, and 3.
The infantile-onset SMA study included 21 patients who had an average age of 6.7 months when the study began. After 12 months of treatment, 41 percent of patients were able to sit independently for more than five seconds. After 23 or more months of treatment, 81 percent of patients were alive without permanent ventilation.
The second randomized, placebo-controlled study evaluated 180 patients with later-onset SMA aged two to 25 years. Patients on Evrysdi saw an average 1.36 increase in their score at the one-year mark, compared to a 0.19 decrease in patients on placebo.
After receiving Fast Track and Priority Review designation, Evrysdi also received Orphan Drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases. Additionally, it was awarded a Rare Pediatric Disease Priority Review Voucher.
Separately, Roche said Evrysdi will be available in the U.S. within two weeks for direct delivery to patients’ homes through Accredo Health Group, an Express Scripts specialty pharmacy. Evrysdi is administered daily at home in liquid form by mouth or by feeding tube.
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