Roche wins new Evrysdi approval for youngest SMA patients – Endpoints News


Roche has just taken the spinal muscular atrophy race up a notch.

Big Pharma has secured another FDA approval for its oral SMA drug Evrysdi, although Tuesday’s green light comes for babies under two months old. This is the first such approval for any SMA drug available for home administration in this infant population.

“Evrysdi’s approval for pre-symptomatic babies is particularly important because early treatment of SMA, before symptoms begin to appear, can help babies achieve motor milestones,” said the lead researcher. of Phase II, Richard Finkel, in a press release.

First approved in 2020 as the first oral drug for SMA, Evrysdi has quickly become a key player in the SMA field and a challenger for Biogen’s Spinraza and Novartis’ Zolgensma. Oral administration probably makes Evrysdi more appealing to patients, since Spinraza is injected into the spine and Zolgensma is given by IV infusion.

Analysts have estimated Evrysdi’s peak sales at around $2 billion, making it a potential blockbuster despite being priced well below competitors. One year of Evrysdi treatment peaks at $340,000 per year, compared to Spinraza’s price of approximately $625,000 to $750,000 in the first year and $375,000 in subsequent years, and Zolgensma’s one-time cost of 2 .1 million.

For infants weighing less than 15 pounds and under the age of two, Evrysdi’s costs are even lower, amounting to less than $100,000 per year. The pricing scale tops out when the child reaches 44 pounds, which puts the average at about $7727.27 per pound per year for each patient.

Falling costs also haven’t stopped Evrysdi from becoming one of Roche’s fastest growing cash engines. In the first quarter of 2022, the drug generated more than $235 million in sales, an increase of 189% compared to the same period last year. Roche said last month that European markets had seen the greatest adoption.

Tuesday’s approval comes from interim results from an open-label, phase II study examining about 25 infants with confirmed diagnoses of SMA who are not yet showing symptoms. Of the six babies evaluable for efficacy so far, all have been able to sit up after a year of treatment, Roche said. Additionally, four of the six could stand, three could walk independently, and all were alive without ventilation after one year.

In addition to this study, dubbed RAINBOWFISH, Roche continues to test Evrysdi in four other clinical trials involving both infants and adults. The drug, licensed from Roche by PTC Therapeutics, is designed to treat SMA by altering the way the SMN2 gene is spliced, resulting in increased levels of functional proteins.


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