Spinal muscular atrophy
While the approval of AveXis’ gene therapy for spinal muscular atrophy has been tainted by the revelation of early data manipulation, the Novartis subsidiary presented some long-term data at a conference that backs up the treatment’s efficacy.
AveXis presented new interim data from the Phase III SPR1NT trial of Zolgensma (onasemnogene abeparvovec-xioi) in spinal muscular atrophy (SMA) Type 1.
Catalyst Pharmaceuticals, Inc., a biopharmaceutical company focused on developing and commercializing innovative therapies for people with rare debilitating, chronic neuromuscular and neurological diseases, announced its support of Spinal Muscular Atrophy Awareness Month.
Novartis Chief Executive Officer Vas Narasimhan is excited about the company’s progress through the first half of 2019 and expects that momentum to continue with a “catalyst-rich” second half of the year.
Now that Novartis has a competing therapy on the market for spinal muscular atrophy (SMA), Biogen is eager to show why it thinks its Spinraza (nusinersen) is superior.
The price of the one-and-done gene therapy treatment is about half of what was initially projected by Novartis.
Genentech, a Roche company, presented data from its FIREFISH clinical trial of risdiplam in Type 1 spinal muscular atrophy (SMA) at the American Academy of Neurology Annual Meeting.
Just as Novartis and AveXis’ gene therapy, Zolgensma (onasemnogene abeparvovec-xioi) for spinal muscular atrophy (SMA) is progressing toward approval, a second patient death was reported.
AveXis, a Novartis company, announced interim data from its Phase III STR1VE clinical trial of Zolgensma (onasemnogene abeparvovec-xioi) in spinal muscular atrophy (SMA) Type 1.
“Think of it as an installment plan that’s then tied to how well the therapy works,” Mark Trusheim, strategic director of the MIT Center for Biomedical Innovation’s NEWDIGS program, told Business Insider. “This would be a car loan but you’ve still got to see if the car is going to work.”
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