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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