Roche nabs a priority review for Evrysdi, hoping to become the first oral treatment for infants with SMA
Roche has been duking it out with Biogen and Novartis ever since bursting into the spinal muscular atrophy space about a year and a half ago with its competitively priced oral treatment Evrysdi. Now the pharma giant is looking to stake its claim in a younger subset of patients — and the FDA has agreed to give it a speedy review.
Regulators have granted priority review to a supplemental NDA for Evrysdi (risdiplam) to treat pre-symptomatic babies under 2 months old with SMA. While the drug is currently approved for adults, children and babies older than 2 months, a new approval here would make it the first at-home treatment available for younger infants. A decision is expected by May 30, according to Roche.
“Treating very young babies with Evrysdi before SMA symptoms arise may help them to achieve milestones such as standing and walking within timeframes typical of healthy infants,” CMO Levi Garraway said in a statement.
Evrysdi — licensed by Roche from PTC Therapeutics — is designed to treat SMA by modifying how the SMN2 gene is spliced, thus increasing functional SMN protein levels in both the central nervous system and peripheral tissues. SMN protein is critical for maintaining healthy motor neurons and movement.
The drug got a quick OK back in 2020, becoming the first oral treatment for SMA. Analysts penciled in a peak sales estimate of around $2 billion. Its competitors, Biogen’s Spinraza and Novartis’ Zolgensma, are administered by injection to the spinal cord and IV infusion, respectively.
“I think that that’s really important. One, just in general, saving people the inconvenience of going to a hospital. I think most people would much rather take something by mouth than have a procedure done, and probably even more important in COVID,” PTC COO Matthew Klein told Endpoints News. “I think people are really not wanting to… leave home, let alone go to medical facilities.”
At an interim analysis in the RAINBOWFISH study, all five patients under 2 months old maintained the ability to swallow and were able to feed exclusively by mouth after 12 months of treatment, according to Roche. Four of those five patients achieved milestones such as standing and walking independently, and all met the HINE-2 motor milestones of head control, sitting upright, rolling and crawling after 12 months.
The drug appeared safe, with no treatment-related serious events reported in any of the babies. Four treatment-emergent side effects were reported out of 12 babies, all of which were resolved or resolving with ongoing Evrysdi treatment. The most common side effects were nasal congestion (33%), cough (25%), teething (25%), vomiting (25%), eczema (17%), abdominal pain (17%), diarrhea (17%), gastroenteritis (17%), papule (17%) and pyrexia (17%).
“The AEs were reflective of the age of the babies rather than the underlying SMA,” Roche said.
From the start, Roche promised to “underwhelm” with Evrysdi’s price compared to competitors. The pharma giant set the price on a scale based on a child’s weight, maxing out when they reach 44 lbs at about 6 years of age. For example, for an infant who weighs 15 lbs and is less than 2 years old, the annual price of Evrysdi would be less than $100,000, a spokesperson told Endpoints.
Meanwhile, Zolgensma has earned the notorious title of the most expensive drug in the world at $2.1 million per patient, and back in 2020, Spinraza cost between $625,000 and $750,000 for the first year and around $375,000 every year after.
Evrysdi’s lower price hasn’t stopped it from reeling in mammoth sales. In Q3 of 2021, the drug netted around $429 million, compared to Zolgensma’s $375 million and Spinraza’s $444 million. Spinraza’s Q3 sales decreased from $494 million in Q3 of 2020, which Biogen attributed to growing competition and the impacts of Covid-19.
Roche shelled out $30 million upfront for the rights to Evrysdi back in 2011, promising up to $460 million in biobucks and up to double-digit royalties on commercial sales. PTC raked in $35 million in milestone payments in Q3 of 2020 and $7.5 million in Q4, according to the company’s year-end report. The drug is currently approved in 70 countries and has been submitted for approval in 31 more.