My heart is heavy today following PTC’s decision to withdraw their NDA submission for Atalauren (Translarna). PTC submitted their package to FDA in 2024, based on FDA’s commitment to review the totality of evidence for Atalauren, a small molecule for individuals with nonsense variants. PTC’s decision is based on recent discussions with FDA during which they shared that based on its review to date, the data in the NDA submission are unlikely to meet the Agency’s threshold of substantial evidence of effectiveness to support approval. Like many of you, I feel the weight of this decision deeply. The journey to finding effective treatments for Duchenne and Becker has been long and arduous. Setbacks like this one are undoubtedly disheartening, particularly for the approximately 130 individuals with Duchenne currently on Atalauren in the U.S., some of whom have been on Translarna for more than 10 years.
As a community, we have worked tirelessly, advocating, fundraising, and participating in clinical trials. We want to acknowledge and thank the brave patients and families who participate in clinical trials. Each of you has demonstrated remarkable resilience and unwavering commitment, and your contributions are invaluable in helping to bring therapies to all in our community. While this news is a setback, it is not the end of the road.
We anticipate further updates from PTC regarding their next steps and future plans, and we thank PTC for their long term commitment to the Duchenne community. PPMD will continue to work closely with researchers, industry partners, and the entire Duchenne community to ensure that every effort is made to bring safe and effective treatments to those who need them.
Thank you for your strength, courage, and dedication. We will navigate this setback together, and continue fighting for every future.
Read PTC’s Letter to the Community here.
The post PTC Therapeutics Provides Regulatory Update on Translarna™ (atalauren) appeared first on Parent Project Muscular Dystrophy.