FDA Approves Evrysdi, Third Drug for Spinal Muscular Atrophy

Spinal muscular atrophy (SMA) is a rare inherited genetic disorder that affects motor neurons. Until recently, outside of patients, their families and clinicians who treated the disease, relatively few people have heard about of SMA.

But that changed with the approval of Spinraza (nusinersen) in 2016 and Zolgensma (onasemnogene abeparvovec-xioi) in 2019 and their price tags.

Zolgensma, a one-time gene therapy treatment, is priced at $2.1 million and shows up on various lists as being the most expensive drug in the world. Spinraza is priced at $750,000 for the first year and $375,000 for treatment in subsequent years. Cumulatively, it may be even more expensive than Zolgensma, depending on how many years a person is treated.

On Friday, the FDA approved a third SMA drug that may kick off a new era of price competition among SMA drugs.

Evrysdi (risdiplam) will be priced so the highest annual cost will be $340,000, a Genentech spokesperson told Endpoints News on Friday. Genentech is marketing the drug in the U.S. and its parent company, Roche, is marketing it elsewhere. In a press release on Friday, Genentech highlighted the fact that it Evrysdi is liquid medicine that can be administered at home by mouth or feeding tube. As the name suggests, it is a daily medication.

Even if the price is lower than Spinraza and Zolgensma, analysts believe that Evrysdi will be a blockbuster with sales of $2 billion, according to Endpoints News.

The press release also noted that one of the two trials (called Sunfish) upon which FDA approval was based is the first and only placebo-controlled trial to include adults with SMA Types 2 and 3 (SMA is grouped into five types).

Evrysdi will delivered be to people’s homes by Accredo, the specialty pharmacy owned by Express Scripts.

Levi Garraway

“Given the majority of people with SMA in the U.S. remain untreated, we believe that Evrysdi, with its favorable clinical profile and oral administration, may offer meaningful benefits for many living with this rare neurological disease,” said Levi Garraway, M.D., Ph.D., the chief medical officer and head of global product development for Genentech.