FDA hands out a quick OK for potential SMA blockbuster risdiplam, giving Genentech and Roche a chance to challenge rivals on the price
US regulators handed Roche and Genentech a big win Friday afternoon, one that has market-shaping potential for its high-priced rivals from Novartis and Biogen.
The FDA has green-lit the companies’ spinal muscular atrophy drug risdiplam, which will be marketed as Evrysdi in the US, for use in patients two months and older. It’s the first SMA drug that can be taken orally, as Biogen’s Spinraza is injected into the spine while Novartis’ Zolgensma is a gene therapy.
Analysts have penciled in a peak sales estimate of around $2 billion for this drug.
“Evrysdi, with its at-home delivery made possible by oral dosing, represents an important and timely advancement for people with SMA,” said Genentech CMO Levi Garraway in a statement emailed to Endpoints News. “During these challenging times, we are extremely proud to bring this groundbreaking medicine to those living with this highly disabling, rare neurological condition.”
Risdiplam will be marketed in the US by Genentech and globally by Roche should it attain further approvals. The biotech developed risdiplam with the SMA Foundation and partner PTC Therapeutics.
Friday’s announcement came ahead of schedule, as the PDUFA date on risdiplam had been slated for August 24.
As for the cost, Evrysdi is priced significantly lower than its SMA competitors. The price maxes out at $340,000 annually, a Genentech spokesperson told Endpoints News, and is scaled on a patient’s weight. The cost for an infant weighing 15 pounds who is less than two years old would be under $100,000, the spokesperson added.
The pricing scale caps when the child reaches 44 pounds, which puts the average at about $7727.27 per pound per year for every patient.
Roche CEO Bill Anderson said early this year that the plan was to “underwhelm with our pricing” and they seem to have done so, and more. Underwhelming didn’t prove very challenging — Zolgensma has the dubious designation of most expensive drug in the world at $2.1 million per patient, while Spinraza costs $750,000 for the first year and then $375,000 every subsequent year.
The reasoning behind the weight-based scale is due to how infants metabolize drugs differently than older individuals, PTC CEO Stuart Peltz told Endpoints, adding “the strategy is there’s a large number of patients who are untreated, and I think Roche’s expectation is to get it to the patients as rapidly as possible.”
Evrysdi was approved based on two clinical trials that tested efficacy in various ages and severities, and the FDA handed the drug quite the broad label. Previously only Spinraza had been approved to treat SMA in adults and Peltz said the development teams focused on a broad trial population in order to serve patients that had previously been underrepresented. No approved treatments for SMA existed before 2016.
The Covid-19 pandemic has also thrown a wrinkle into Evrysdi’s launch given the drug’s ease of use at home. While the other therapies involve going out to receive injections, Evrysdi is a liquid ingested once a day orally and Peltz said he believes this accessibility will make the drug more preferable for those still too nervous to venture outdoors frequently.
Baird analyst Brian Skorney largely agreed with that assessment, writing to investors, “With the fear of COVID on everyone’s mind, we think it puts Evrysdi in a marketable advantage over Spinraza.” That, combined with Evrysdi’s significantly lower cost, results in the drug “putting significant competitive pressure” on Spinraza, which garnered $2.1 billion in 2019.