FDA extends PDUFA date for review of risdiplam to treat spinal muscular atrophy.- Genetech/Roche

Genentech, a member of the Roche Group announced that the FDA has extended the Prescription Drug User Fee Act (PDUFA) date for its review of the New Drug Application (NDA) of risdiplam with a decision expected by 24 August 2020.

The extension is a result of the recent submission of additional data by Genentech, including data from the pivotal SUNFISH Part 2 study, in close collaboration with the FDA. These data were recently presented at the 2nd International Scientific and Clinical Congress on Spinal Muscular Atrophy. In November 2019, the FDA granted Priority Review for risdiplam with a decision for approval expected by 24 May 2020.

In February 2020, based on discussions with the FDA, Genentech submitted additional data which could help ensure access to risdiplam for a broad range of people living with the condition, if approved. This included 12-month efficacy and safety data from the pivotal SUNFISH Part 2 study (n=180), the only placebo-controlled study ever undertaken in people aged 2-25 years with Type 2 or 3 SMA. Given the volume of additional data submitted, the FDA requires more time for review..