PTC Therapeutics Withdraws Translarna NDA After FDA Feedback on Efficacy

PTC Therapeutics, Inc. (NASDAQ: PTCT) announced on February 12, 2026 that it has withdrawn the New Drug Application (NDA) resubmission for Translarna (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy (DMD) following U.S. Food and Drug Administration (FDA) feedback on the application review. The FDA 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 of Translarna.

The Chief Executive Officer stated that the company has worked tirelessly for over two decades to develop a safe and effective therapy for boys and young men affected by nonsense mutation DMD in the U.S. and is disappointed that FDA approval cannot be achieved.

Translarna is an investigational therapy for nonsense mutation Duchenne muscular dystrophy, a rare and serious genetic condition. The drug is a protein restoration therapy designed to enable the formation of a functioning protein in patients with genetic disorders caused by a nonsense mutation. A nonsense mutation is an alteration in the genetic code that prematurely halts the synthesis of an essential protein.

Duchenne muscular dystrophy primarily affects males and is a rare and fatal genetic disorder that results in progressive muscle weakness from early childhood and leads to premature death in the mid-20s due to heart and respiratory failure. It is a progressive muscle disorder caused by the lack of functional dystrophin protein. Dystrophin is critical to the structural stability of all muscles, including skeletal, diaphragm, and heart muscles. Patients with Duchenne can lose the ability to walk (loss of ambulation) as early as 10 years old, followed by loss of the use of their arms. Duchenne patients subsequently experience life-threatening lung complications, requiring the need for ventilation support, and heart complications in their late teens and 20s.

The decision marks a meaningful shift for the company and raises fresh questions around its rare disease pipeline and regulatory path. For a rare disease company that leans heavily on a small portfolio, the withdrawal of a potential near term US product opportunity matters for long term revenue mix and bargaining power with payers compared with peers like Sarepta Therapeutics, BioMarin and Vertex Pharmaceuticals, which each have multiple commercial or late stage rare disease assets.

Shares of PTC Therapeutics were at $69.17, up 39.9% over the past year and 54.7% over three years, while the year to date return of a 9.9% decline highlights recent pressure as the market reassesses risk and expectations. The decision keeps the focus squarely on broader rare disease launches such as Sephience, which has been highlighted as a key driver for building a multi product, multi market platform.

PTC is a global biopharmaceutical company dedicated to the discovery, development and commercialization of clinically differentiated medicines for children and adults living with rare disorders. PTC is advancing a robust and diversified pipeline of transformative medicines as part of its mission to provide access to best-in-class treatments for patients with unmet medical needs.