On the heels of Phase 3 data showing muscle improvement in the rare neuromuscular disease spinal muscular atrophy, Scholar Rock raised $300 million in a stock offering. U.S. and European regulatory submissions for the drug are now planned for early 2025.
Novartis is paying Voyager Therapeutics $100 million up front to collaborate on gene therapies for Huntington’s disease and spinal muscular atrophy. The deal builds on a relationship the companies started in 2022.
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The two deaths reported following treatment with Novartis’s Zolgensma mark the first fatal cases of acute liver failure associated with the gene therapy. Liver complications are a known side effect risk of Zolgensma and other genetic medicines that are delivered by engineered viruses.
Roche drug Evrysdi, one of three FDA-approved treatments for spinal muscular atrophy, is now approved for treating those younger than 2 months of age. The regulatory decision puts the oral drug on equal footing with SMA drugs from Novartis and Biogen.
Neuroscience drug developer Biohaven Pharmaceuticals is expanding its pipeline to include epilepsy and spinal muscular atrophy. The drug developer is acquiring Channel Bioscience and licensing global rights to a Phase 3-ready drug from Bristol Myers Squibb.
Analysts noted that Evrysdi would be priced significantly lower than Biogen's Spinraza or Novartis' Zolgensma. While this will provide a market advantage, it would also yield lower per-patient revenues.
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The company plans to enroll 60 patients who have received its drug, Spinraza, following suboptimal response to Novartis' Zolgensma, which was approved last year. The trial, RESPOND, is anticipated to start enrolling in the first quarter of 2021, pending regulatory approval.
An analyst wrote that while the delay for risdiplam - prompted by Roche's submission of additional data - is unfortunate, the timing of the pivotal trials and the patient population guarantee a broad label for the drug, making the company's decision prudent.
The agency gave priority review to Roche's risdiplam, developed for SMA in infants and adults. The drug would potentially compete against Biogen's Spinraza and Novartis' gene therapy, Zolgensma.
The hold places a pause on enrollment in the high-dose cohort of the company's Phase I study of intrathecal AVXS-101, a different administration for the marketed therapy Zolgensma. It does not affect the marketed product and is unrelated to an early data manipulation scandal.
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The company posted strong sales for Zolgensma, which the FDA approved five months ago for treating infants with spinal muscular atrophy. Strong and growing sales were also reported for the breast cancer drug Piqray and the CAR-T cell therapy Kymriah.
Interim Phase I/II data for Zolgensma showed improvements in children aged 2 to less than 5 with spinal muscular atrophy. The therapy received FDA approval in May for infants younger than 2.
The Aug. 23 document from AveXis, posted on the FDA website Tuesday, fingered "two AveXis senior executives" who had been terminated. Brothers Brian and Allan Kaspar were ousted from AveXis last month, though the former denied any wrongdoing.
Novartis subsidiary AveXis is presenting Phase III data at a European pediatric neurology conference on the therapy, Zolgensma, which won approval in May for spinal muscular atrophy in infants.
The Wall Street Journal reported that the agency was focusing on the two-month delay between the March 14 discovery of data manipulation by Novartis' AveXis unit regarding the therapy Zolgensma and its May 15 launch of a formal inquiry into the matter.