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07.04.2020 22:05:00

Roche provides regulatory update on risdiplam for the treatment of spinal muscular atrophy (SMA)

Basel, 07 April 2020 - Roche (SIX: RO, ROG; OTCQX: RHHBY), today announced that the U.S. Food and Drug Administration (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 August 24, 2020. The extension is a result of the recent submission of additional data by Roche, 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 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, 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. “We strongly believe in the potential of risdiplam as a new therapeutic option and recognize that unmet need remains in the treatment of SMA,” said Levi Garraway, M.D., Ph.D., chief medical officer and head of Global Product Development. “We are working closely with the FDA to support the review of risdiplam. Our goal is to bring this therapy to infants, children and adults living with SMA as quickly as possible." As part of our continued commitment to people living with SMA, Roche is pleased to announce that we have also submitted in Brazil, Chile, Indonesia, Russia, South Korea and Taiwan. Filing in China is imminent and we are currently on track to submit a Marketing Authorization Application (MAA) to the European Medicines Agency in mid-2020 as well as other international markets. Roche leads the clinical development of risdiplam, an investigational, orally administered survival motor neuron-2 (SMN2) splicing modifier for SMA, as part of a collaboration with the SMA Foundation and PTC Therapeutics. Risdiplam is being studied in a broad clinical trial programme in SMA, with patients ranging from birth to 60 years old, and includes patients previously treated with other SMA-targeting therapies. The clinical trial population is designed to represent the broad, real-world spectrum of people living with this disease with the aim of ensuring access for all appropriate patients.
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