FDA Approves Roche’s Spinal Muscular Atrophy Drug

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FDA has approved Evrysdi (risdiplam) for treating spinal muscular atrophy in adults and children.

On Aug. 10, 2020, Roche announced that FDA approved Evrysdi (risdiplam), a new drug for treating spinal muscular atrophy (SMA). The drug is indicated for adults and children aged two months and older. Evrysdi is a liquid that can be administered at home by mouth or with a feeding tube.

The drug works by increasing production of the survival of motor neuron (SMN) protein, which is found throughout the body and maintains healthy motor neurons and movement, Roche stated in a company press release. The clinical development of Evrysdi is being conducted by Roche in collaboration with the SMA Foundation and South Plainfield, NJ-based pharmaceutical company PTC Therapeutics. Evrysdi will be available in the United States for direct delivery to patients’ homes.

“Given the majority of people with SMA in the US remain untreated, we believe Evrysdi, with its favorable clinical profile and oral administration, may offer meaningful benefits for many living with this rare neurological disease,” said Levi Garraway, MD, PhD, Roche’s chief medical officer and head of global product development, in the press release.

“Throughout their lives, many people with SMA may lose their ability to perform critical movements, which can impact the ability to independently participate in aspects of daily life and even be life altering,” said Kenneth Hobby, president of Cure SMA, in the press release. “The approval of Evrysdi is an eagerly awaited milestone for our community. We appreciate Genentech/Roche’s commitment to reflecting the full scope of the real-world SMA population in their clinical trial program and developing a treatment that can be administered at home.”


The European Medicines Agency (EMA) granted risdiplam, the active ingredient in Evrysdi, priority medicines designation in 2018, and both FDA and EMA granted it orphan drug designation in 2017 and 2019, respectively. Risdiplam has also been filed in Brazil, Chile, China, Indonesia, Russia, South Korea, and Taiwan. Roche expects a decision soon from the EMA on its European market application for the drug.

Source: Roche