The SMA Foundation’s partners, Roche and PTC Therapeutics, announced positive data from the pivotal Part 2 of FIREFISH, a study evaluating risdiplam in infants aged 1-7 months with Type 1 SMA. The study met its primary endpoint of a statistically significant proportion of infants sitting without support for at least five seconds after one year of treatment. Risdiplam is an investigational, orally administered SMN2 splicing modifier that increases SMN protein in the central nervous system and peripheral tissues of the body. No treatment related safety findings leading to withdrawal have been seen in any risdiplam trial to date. Data from the FIREFISH study will be presented at an upcoming medical congress.
Positive results from Part 2 of SUNFISH, a study evaluating the efficacy and safety of risdiplam in people aged 2-25 years with Type 2 or 3 SMA, were announced in November 2019. Also in November 2019, the U.S. Food and Drug Administration (FDA) granted Priority Review for risdiplam with a decision for approval by May 24, 2020.