Roche has won approval from the US Food and Drug Administration (FDA) for its spinal muscular atrophy (SMA) drug risdiplam under the brand name Evrysdi.
Evrysdi has been approved for the treatment of SMA in adults and children two months of age and older, following positive results across two clinical trials of different ages and levels of disease severity, including SMA type 1, 2 and 3.
The approval is based on data from two clinical studies – FIREFISH in symptomatic infants aged two to seven months and SUNFISH in children and adults aged two to 25 years old.
In the FIREFISH study, 41% of infants receiving Evrysdi achieved the ability to sit without support for at least five seconds, measured by the Bayley Scales of Infant and Toddler Development Third Edition (BSID-III).
In addition to improving motor function scores, 90% of infants treated with Roche’s drug were alive without permanent ventilation at 12 months of treatment, and reached 15 months of age or older.
Positive results were also observed in the SUNFISH study, wherein children and adults treated with Evrysdi experienced a clinically meaningful and statistically significant improvement in motor function at 12 months compared to placebo.
SMA is a muscle-wasting disease that affects around one in 6,000 to 10,000 live births and is a leading cause of genetic death in infants. Evrysdi becomes the third drug approved to treat the condition and is the first that can be administered orally.
The first drug to reach the market for SMA was Biogen’s Spinraza (nusinersen) back in 2016. It is also indicated for use in children and young adults with all forms of the disease, including the most severe type 1 variant.
Following Spinraza to approval was Novartis’ one-shot intravenous gene therapy Zolgensma (onasemnogene abeparvovec) which is approved to treat children with SMA under the age of two years old.
With benefit in younger, type 1 patients as well as in older patients with SMA, Roche’s drug is primed to take on both Spinraza and Zolgensma, with the added benefit that it can be taken at home.
Roche has priced Evrysdi by patient weight, with a maximum cost of $340,000 per year, which is much less than its competitors. Sprinraza’s list price is $750,000 in the first year and $375,000 thereafter, while Zolgensma carries a price-tag of $2.1m – the most expensive drug in the world.