PTC Announces Regulatory Update on Risdiplam for Spinal Muscular Atrophy

PTC Therapeutics announced 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 to August 24, 2020. Roche recently submitted additional data including comprehensive data from SUNFISH part 2 to help provide access to risdiplam for a broad range of people living with spinal muscular atrophy (SMA), triggering this extension. The FDA has informed Roche that the review team is working expeditiously to complete their review of the application as quickly as possible. The FDA has also indicated to Roche 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," said Stuart W. Peltz, Ph.D., 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."

In order to further support broad, global access to risdiplam for people living with SMA, Roche has submitted applications for approval in Indonesia, Taiwan, China, 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 mid-2020.

In November 2019, the FDA granted Priority Review for risdiplam. Risdiplam has the broadest clinical trial program being evaluated to treat SMA, with patients ranging from birth to 60 years old, and includes patients previously treated with SMA-targeting therapies, including approved treatments. The clinical trial population represents the diverse, real-world spectrum of people living with this disease with the aim of ensuring access for all appropriate patients. To date, more than 400 patients 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 SMA program is a collaboration between PTC, the SMA Foundation, and Roche.

Spinal muscular atrophy (SMA) is a severe, inherited, progressive neuromuscular disease that causes devastating muscle atrophy and disease-related complications. It is the most common genetic cause of infant mortality and one of the most common rare diseases, affecting approximately one in 11,000 babies. SMA leads to the progressive loss of nerve cells in the spinal cord that control muscle movement. Depending on the type of SMA, an individual's physical strength and their ability to walk, eat or breathe can be significantly diminished or lost.

SMA is caused by a mutation in the survival motor neuron 1 (SMN1) gene that results in a deficiency of SMN protein. SMN protein is found throughout the body and increasing evidence suggests SMA is a multi-system disorder and the loss of SMN protein may affect many tissues and cells, which can stop the body from functioning.

Risdiplam is an investigational survival motor neuron2 (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. Risdiplam is being studied in a broad clinical trial program 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 represents the broad, real-world spectrum of people living with this disease. The risdiplam clinical development program was designed with the aim of enabling access for all appropriate patients.

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