The approval was based on results from the SELUTION4ISR trial, a single blind, randomized, non-inferiority trial conducted across 48 centers in the US, Europe, Canada, and Brazil. Investigators enrolled patients >18...
Approval is based on 36-week data from BRAVE-AA-PEDS, in which 42% of patients on the 4 mg dose achieved a SALT score of 20 or less, compared with 5% on placebo.
The FDA approved once-daily oral zilurgisertib for FOP in patients aged 12 years and older after the phase 2 PROGRESS trial showed reduced new heterotopic ossification vs placebo.