The US 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 (Roche; Genentech, San Francisco, CA) to August 24, 2020. Additional data was submitted, including comprehensive data from SUNFISH part 2 (NCT02908685), to support providing access to risdiplam for a broad range of people living with spinal muscular atrophy (SMA), causing the extension.
In November 2019, the FDA granted Priority Review for risdiplam. Risdiplam has the broadest clinical trial program being evaluated to treat SMA, with participants ranging from birth to the age of 60 years, and includes participants previously treated with SMA-targeting therapies, including approved treatments. The clinical trial population represents the diverse, real-world spectrum of individuals living with SMA to ensure access for all appropriate individuals. More than 400 participants have been treated with risdiplam across all studies to date, with no treatment-related safety findings leading to study withdrawal in any risdiplam trial.
The FDA’s review team is working to complete their review of the application as soon as possible. The FDA has also indicated that no substantive review issues have been identified to date.
"We are encouraged that the FDA has no substantive review issues. Their interest in the additional results from the clinical studies demonstrating risdiplam's activity supports our goal of enabling access to this important therapy for all SMA patients," stated Stuart W. Peltz, PhD, chief executive officer, PTC Therapeutics. "Enabling access to a home-administered oral therapy for a broad patient population is critically important and we look forward to the FDA living up to its commitment to review the application as quickly as possible."
To further support broad, global access to risdiplam for people living with SMA, Roche has submitted applications for approval in Indonesia, Taiwan, Chile, Brazil, South Korea, and Russia and the submission of the filing in China is imminent. The submission of a marketing authorization application (MAA) to the EMA and filings in other international markets remain on track for midyear 2020.
Stephen M. Gollomp, MD, and Paul G. Mathew, MD, DNBPAS, FAAN, FAHS
Claire Smyth, BSc; David Roberts, BSc; and Kenneth Monaghan, PhD
James Geyer, MD, and Paul Cox