25 November 2019 - Filing submission includes 12-month data from pivotal FIREFISH and SUNFISH trials in a broad population of people living with Types 1, 2 or 3 SMA.
Roche today announced that the U.S. FDA has accepted the new drug application and granted priority review for risdiplam, an investigational survival motor neuron-2 (SMN-2) splicing modifier for SMA. Risdiplam is designed to increase and sustain SMN protein levels both throughout the central nervous system and peripheral tissues of the body.
The FDA is expected to make a decision on approval by 24 May 2020.