Fresh trial data fortify position of Roche's oral therapy in spinal muscular atrophy battleground
With an FDA decision date looming, Roche on Wednesday unveiled positive pivotal data on its blockbuster-bound oral spinal muscular atrophy (SMA) drug in patients with the most severe form of the muscle-wasting disease.
The FDA is set to make its decision on the therapy, risdiplam, by May 24. It is expected to compete with Biogen’s Spinraza and Novartis’ Zolgensma.
Partnered with PTC Therapeutics, the Roche drug was tested in 41 patients aged 1-7 months with type 1 SMA, a rare genetic muscle-wasting disease. The trial, dubbed FIREFISH, measured efficacy via the proportion of infants sitting without support after 12 months of treatment, and longer.
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