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ARTICLE | Clinical News

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

January 23, 2020 7:35 PM UTC
Updated on Jan 24, 2020 at 12:47 AM UTC

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. ...