Roche uncorks new long-term data as SMA rivalry heats up
Leading up to a regulatory decision in May that could give Roche a big leg up on Biogen and Novartis in the spinal muscular atrophy space, the Swiss pharma giant is hoping some new data will bolster its case.
Roche uncorked some long-term efficacy and safety data for its blockbuster SMA drug Evrysdi (risdiplam) on Wednesday, while also offering another peek at the interim results for its RAINBOWFISH study in newborns under 2 months old.
“In treating people with SMA, our aim is to enable or preserve their independence and patients in the SUNFISH study reported continuous improvement or stabilisation in the level of help needed for daily living,” MDUK Oxford Neuromuscular Centre professor Laurent Servais said in a news release.
The new data come just a couple months ahead of an expected decision for Evrysdi to treat pre-symptomatic babies younger than 2 months. A yes here would make it the first at-home treatment available for younger infants.
In the SUNFISH study — which enrolled patients between 2 and 25 years old with types 2 or 3 SMA — patients were able to maintain their increase in Motor Function Measure 32 (MFM-32) total score from baseline (previously observed at one year) through year three, according to Roche. They were also able to sustain increases in Revised Upper Limb Module (RULM) and Hammersmith Functional Motor Scale Expanded (HFMSE) total scores between years one and three.
Patients also reported fewer side effects as time went on. The overall rate of adverse events decreased over three years, and investigators noted a trend toward a lower rate of serious adverse events in year three, Roche said. Overall, those side effects were reflective of underlying disease, and no treatment-related adverse events led to withdrawal from the study, according to the company.
“We are pleased that these long-term results further reinforce the safety and efficacy of Evrysdi and it is especially encouraging to see that adverse events decreased over time,” CMO Levi Garraway said in the release.
As for RAINBOWFISH, the latest interim results suggest that the majority of babies treated with Evrysdi for at least a year were able to stand and walk within timeframes typical of healthy babies.
From the abstract presented this week at the Muscular Dystrophy Association conference:
Efficacy data from infants receiving risdiplam for ?12 months (n=5) demonstrated that these infants reached a Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders score of ?60 and maintained swallowing and feeding abilities. We will report updated baseline demographics and safety data in enrolled infants and efficacy data in infants who have received risdiplam for ?12 months.
Roche gave PTC Therapeutics $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. The drug works 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.
Evrysdi first cleared the FDA back in 2020, becoming the first oral treatment for SMA. Its competitors, Biogen’s Spinraza and Novartis’ Zolgensma, are administered by injection to the spinal cord and IV infusion, respectively. But route of administration isn’t the only thing setting Evrysdi apart from rivals.
From the start, Roche promised to “underwhelm” with Evrysdi’s price compared to competitors. The pharma 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. So for an infant who weighs 15 lbs and is less than 2 years old, for example, the annual price of Evrysdi would be less than $100,000, a spokesperson told Endpoints News back in January. Zolgensma, on the other hand, earned itself the notorious title of most expensive drug in the world at $2.1 million per patient.
Evrysdi raked in more than $639 million in 2021 (602 million CHF), compared to just over $1.9 billion for Spinraza and $1.35 billion for Zolgensma.
As the weather heats up for spring, so might Roche’s rivalry with Biogen and Novartis. The FDA granted Roche a priority review for its supplemental NDA in the newborn group back in January, and the company said at the time that it expects a decision by May 30.