Risdiplam hits again in FIREFISH, setting up oral option for SMA patients

As risdiplam inches closer to the market in spinal muscular atrophy, the oral candidate notched another win, hitting the primary endpoint in the Phase II/III FIREFISH study and setting up a new alternative for SMA patients that could be more convenient and at least equally effective.

On Thursday, Roche (SIX:ROG; OTCQX:RHHBY) and partner PTC Therapeutics Inc. (NASDAQ:PTCT) said that risdiplam met the primary endpoint in the second portion of the FIREFISH study. The trial enrolled infants age 1-7 months; the primary endpoint was sitting unassisted for at least five seconds at 12 months after initial treatment. ...