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Roche's Evrysdi Approved By FDA For Treatment Of Spinal Muscular Atrophy

fdaapprove aug10 lt

Roche (RHHBY) said the U.S. FDA has approved the company's Evrysdi (risdiplam) for the treatment of spinal muscular atrophy in adults and children 2 months of age and older. The company noted that Evrysdi is the first and only medicine for spinal muscular atrophy that can be taken at home. It 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 a survival of motor neuron 2 splicing modifier designed to treat spinal muscular atrophy caused by mutations in chromosome 5q that lead to SMN protein deficiency. In two clinical trials, Evrysdi improved motor function in patients at various levels of disease severity, including Types 1, 2, and 3. Evrysdi is administered daily at home in liquid form by mouth or by feeding tube.

Roche leads the clinical development of Evrysdi as part of a collaboration with the SMA Foundation and PTC Therapeutics. Roche noted that a Marketing Authorization Application submission to the EMA for Evrysdi is imminent. Risdiplam has been filed in Brazil, Chile, China, Indonesia, Russia, South Korea, and Taiwan.

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