32. Evrysdi

Evrysdi
Unlike Spinraza, which is injected, and Zolgensma, which is given by intravenous infusion, Evrysdi is a liquid drug delivered by mouth or feeding tube. (Roche)

Active ingredient: Risdiplam
Disease: Spinal muscular atrophy
Peak sales estimate: $2.5 billion to $3 billion
Approved: Aug. 7
Company: Roche

The scoop: Roche’s Evrysdi is the third treatment for spinal muscular atrophy to hit the market, trailing Biogen’s Spinraza and Novartis’ gene therapy Zolgensma. But it has a couple tricks up its sleeve that could give it an edge over the competition. Unlike Spinraza, which is injected, and Zolgensma, which is given by intravenous infusion, Evrysdi is a liquid drug delivered by mouth or feeding tube. It can be given at home, which would be more convenient for patients and their caregivers, especially during the COVID-19 pandemic. And although Evrysdi is costly at up to $340,000 per year, its price depends on a patient’s weight. For some patients, the price could be under $100,000 a year, compared to $750,000 for the first year of Spinraza and $2.125 million for Zolgensma, which is intended to be a one-time treatment. Spinraza’s list price drops to $375,000 a year for the second and subsequent years of treatment. The approval is based on two phase 3 studies—one in infants and one in children and adults aged 2 to 25—that showed the drug improved patients’ motor function. – Amirah Al Idrus

32. Evrysdi