Spinal muscular atrophy
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.”
Using a calculation, ICER believes the Novartis gene therapy for Spinal Muscular Atrophy will be more cost effective than Biogen’s Spinraza.
Novartis’ gene therapy treatment for spinal muscular atrophy could see approval as early as May of this year after the U.S. Food and Drug Administration granted AVXS-101, now dubbed Zolgensma, priority review.
PTC Therapeutics announced interim data from Part 1 of its open-label clinical trials, FIREFISH and SUNFISH, of risdiplam for Type 1, 2 and 3 spinal muscular atrophy in collaboration with Genentech.
PTC Therapeutics presented updated interim clinical data from Part 1 of its FIREFISH clinical trial of risdiplam (RG7916) in babies with Type 1 Spinal Muscular Atrophy.
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