April 9, 2020 / BIOON / -- Roche recently announced that the U.S. Food and Drug Administration (FDA) has extended the target date of prescription drug user fee Act (PDUFA) of risdiplam new drug application (NDA) for three months, and is expected to make a decision on August 24, 2020. Risdiplam is a splicing modifier of motor neuron survival gene 2 (SMN2) for the treatment of spinal muscular atrophy (SMA). The extension of the review period was due to the results of additional data recently submitted by Roche, including data from the key sunfish Part 2 study, which worked closely with the FDA. These data were recently published at the second International Congress of spinal muscular atrophy.
In November 2019, FDA granted risdiplam NDA a priority review and designated the target date of PDUFA as May 24, 2020. In February 2020, according to discussions with the FDA, Roche submitted additional data that, if approved, would ensure access to risdiplam treatment for a wide range of SMA patients. This included 12-month efficacy and safety data from the key success part 2 study (n = 180), the only placebo-controlled study of type 2 or type 3 SMA patients aged 2-25. Given the amount of additional data submitted, FDA needs more time to review.
Dr Levi Garraway, Roche's chief medical officer and head of global product development, said: "we firmly believe in the potential of risdiplam as a new treatment option and recognize that there are still unmet needs for SMA treatment. We are working closely with the FDA to support the review of risdiplam. Our goal is to bring this treatment to infants, children and adults with SMA as soon as possible. As part of its ongoing commitment to SMA patients, Roche is pleased to announce that it has also filed applications in Brazil, Chile, Indonesia, Russia, South Korea and Taiwan. The Chinese mainland's filing is coming soon, and the company is now planning to submit its listing application to the European Medicines Agency (EMA) and other international markets as planned in mid 2020.