PTC Therapeutics has announced positive results from part 2 of SUNFISH demonstrating that the study met its primary endpoint of change from baseline after 1 year of treatment with risdiplam compared to placebo as measured by the Motor Function Measure 32, a scale monitoring severity and progression of fine and gross motor function in patients with a neuromuscular disease such as spinal muscular atrophy (SMA). Risdiplam has been well tolerated and no treatment-related safety findings leading to withdrawal have been seen in any risdiplam trial to date. Data from part 2 of the SUNFISH study will be presented at an upcoming medical congress.
"We are very pleased with the results of the SUNFISH study and are excited to move one step closer to bringing risdiplam globally to all patients living with SMA. The SUNFISH trial shows that risdiplam continues to demonstrate its disease-modifying properties and compelling safety profile," said Stuart W. Peltz, Ph.D., Chief Executive Officer of PTC Therapeutics. "The results from the study will be shared first with regulators globally and then will be presented at a SMA conference early next year. We believe that risdiplam has the potential to enter the market with a best-in-class profile for patients with all SMA types. We are particularly grateful to the SMA community, the patients and investigators who participated in the trials."
Subscribe to our e-Newsletters
Stay up to date with the latest news, articles, and events. Plus, get special offers
from American Pharmaceutical Review – all delivered right to your inbox! Sign up now!
Risdiplam (RG7916), is an investigational, oral, first-in-class, mRNA splicing modifier for the treatment of SMA. SUNFISH is a double‐blind, two‐part, placebo‐controlled trial. Part 1 enrolled patients with type 2 or 3 SMA to evaluate the safety, tolerability, and PK/PD of several risdiplam dose levels. The pivotal SUNFISH part 2, in non‐ambulant patients with Type 2 or 3 SMA, evaluated safety and efficacy of the risdiplam dose level selected from part 1 for 24 months, followed by an open label extension. Patients from 2-25 years of age were enrolled in the study, representing the broad real-world spectrum of patients living with SMA. The SMA program is a collaboration between PTC, the SMA Foundation, and Roche.
Spinal muscular atrophy (SMA) is a genetic neuromuscular disorder that is the leading genetic cause of mortality in infants and toddlers caused by a missing or defective survival of motor neuron 1 (SMN1) gene, which results in reduced levels of SMN protein. The homologous SMN2 gene is predominantly spliced to a truncated mRNA, and only produces small amounts of functional SMN protein. Insufficient levels of SMN protein are responsible for the loss of motor neurons within the spinal cord leading to muscle atrophy and death in its most severe form. It is estimated that this devastating disease affects 1 in every 11,000 children born.
Risdiplam is an investigational medicine being studied in a broad range of patients with SMA from birth to 60 years of age. It is designed to provide sustained increase in SMN protein centrally and peripherally through daily dosing and is being evaluated for its potential ability to help the SMN2 gene produce more functional SMN protein throughout the body. Risdiplam is also being studied in a clinical trial for patients with type 1 SMA, called FIREFISH, in pre-symptomatic babies, RAINBOWFISH and in patients who have been in previous clinical trials for SMA, JEWELFISH.