Weeks after FDA delay, Roche, PTC unveil another batch of positive risdiplam data in severe SMA patients
While the FDA has decided it needs another quarter to review Roche and PTC Therapeutics’ oral SMA therapy, its makers are reporting a steady drumbeat of new positive data. On Tuesday, the companies unveiled one-year data in patients with the most severe form of the disease from a key trial.
The FDA is set to make its decision on the therapy, risdiplam, by August. It is expected to compete with Biogen’s Spinraza and Novartis’ Zolgensma.
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