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FDA Approves First Oral Treatment for Spinal Muscular Atrophy

By Cory Renauer – Aug 7, 2020 at 2:22PM

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Roche and PTC Therapeutics will challenge Novartis and Biogen with competitive pricing for Evrysdi.

Treatment options for nearly everyone born with a lethal muscle-wasting disorder improved on Friday. The U.S. Food and Drug Administration approved Evrysdi from PTC Therapeutics (PTCT -5.36%) and Roche (RHHBY 1.00%), making it the third treatment approved for patients with spinal muscular atrophy (SMA) and the first option that patients can take at home.

Evrysdi priced to compete 

Evrysidi, formerly known as risdiplam, is an easy-to-swallow treatment that allows SMA patients to produce functional copies of the survival motor neuron protein they lack. Roche and PTC Therapeutics set the list price of their new rare disease drug at a relatively modest $340,000 per year to compete with already approved treatments from Biogen (BIIB 1.83%) and Novartis (NVS 1.31%)

Blister packs of medication next to a stamp that reads "FDA Approved."

Image source: Getty Images.

Biogen's Spinraza is a small infusion that must be injected into the back to reach cerebrospinal fluid. Spinraza, the first treatment for SMA, has a list price of $750,000 in the first year and then $375,000 for each year after that. The annual cost covers three maintenance doses spread four months apart.

The FDA approved Evrysdi for the treatment of SMA patients with any level of disease severity, but Roche could have a hard time competing with Novartis' Zolgensma, which is intended to treat patients with SMA type 1, the most severe form of the disease. At $2.125 million per dose, Zolgensma's the most expensive drug Americans have ever seen -- but this gene therapy only requires a single administration. Patients who receive Evrysdi and Spinraza will require regular doses of their treatments for the rest of their lives.

Cory Renauer has no position in any of the stocks mentioned. The Motley Fool owns shares of and recommends Biogen. The Motley Fool has a disclosure policy.

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