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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