Priority review to risdiplam for spinal muscular atrophyFebruary 6, 2020
The US FDA has granted Priority Review for risdiplam, an investigational survival motor neuron-2 (SMN-2) splicing modifier for spinal muscular atrophy (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 May 24, 2020.
The risdiplam NDA submission incorporates 12-month data from the dose-finding Part 1 sections of the FIREFISH and SUNFISH pivotal studies, as well as data from the confirmatory Part 2 of SUNFISH. FIREFISH is an open-label, two-part pivotal clinical trial in infants with Type 1 SMA. Part 1 was a dose-escalation study in 21 infants aged one to seven months.
If approved, risdiplam, an orally administered liquid, would be the first at-home administered medicine for people living with SMA.
Risdiplam is currently being evaluated in four multicentre trials in people with SMA.