Babies that have spinal muscular atrophy now have an at-home treatment option that can help extend their lives.
The FDA’s approval of Roche’s Evrysdi opens the oral drug up for infants younger than 2 months old. It was originally approved in August of 2020 for adults and children older than 2 months.
Evrysdi, which was developed by PTC Therapeutics and licensed to Roche/Genentech, is one of three drugs available for SMA.
Biogen’s Spinraza, which was endorsed in 2016, is taken through spinal injection. And Novartis’ gene therapy Zolgensma, which made headlines in 2019 when it was blessed by the FDA and became the most expensive drug in history at $2.1 million a pop, is taken by a one-and-done infusion.
Before Tuesday’s approval, Spinraza and Zolgensma were the only options for infants younger than 2 months.
Roughly half of the pre-symptomatic babies who have been treated with Evrysdi have been able to walk independently within 12 months. Results from the Rainbowfish trial also showed 100% of patients could sit up within 12 months and 67% could stand.
“Early treatment of SMA, before symptoms start to arise, can help babies to achieve motor milestones,” Richard Finkel, M.D., the principal investigator of the trial and Director of the Experimental Neuroscience Program at St. Jude Children’s Research Hospital, said in a release. “With the inclusion of SMA in newborn screening programs, this approval provides the opportunity to start treating at home with Evrysdi soon after the diagnosis is confirmed.”
The nod in infants is more bad news for Biogen, which has seen sales of Spinraza plateau. After generating $2.1 billion in 2019 and 2020, Spinraza’s revenue came in at $1.9 billion last year. Meanwhile, Zolgensma registered sales of $1.4 billion last year.
Industry watchers have also predicted that Evrysdi is more of a threat to Spinraza because both therapies require chronic treatment, while Zolgensma is a one-time gene therapy.
Since its launch in 2020, Evrysdi has been gradually growing its sales. In the first quarter, Evrysdi almost tripled global sales year over year to 226 million Swiss francs ($235 million). By contrast, Spinraza brought in $472 million in the first quarter, a fall from $520 million the same period last year.