FDA extends review for Roche SMA treatment

April 7, 2020

The U.S. Food and Drug Administration has extended the Prescription Drug User Fee Act (PDUFA) date for its review of the New Drug Application (NDA) of Roche's risdiplam with a decision expected by August 24, 2020. The extension is a result of the recent submission of additional data by Roche, including data from the SUNFISH Part 2 study, in collaboration with the FDA. Data was 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 May 24, 2020. In February 2020, based on discussions with the FDA, Roche submitted additional data which could help ensure access to risdiplam for a broad range of people living with the condition. This included 12-month efficacy and safety data from the 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.

Risdiplam is an investigational survival motor neuron-2 (SMN2) splicing modifier for SMA and is an orally administered liquid. It is designed to durably increase and sustain SMN protein levels both throughout the central nervous system and in peripheral tissues of the body. It is being evaluated for its potential ability to help the SMN2 gene produce more functional SMN protein throughout the body.

Read the full Roche release