Drugs

PTC Yanks Translarna NDA After FDA Signals Data Shortfall

PTC Therapeutics has withdrawn its NDA resubmission for Translarna (ataluren) to treat nonsense mutation Duchenne muscular dystrophy, following feedback from FDA indicating the application was unlikely to meet the agency’s evidentiary standard for approval.

The Warren, N.J.-based drugmaker said FDA informed the company during its ongoing review that the data package did not appear sufficient to satisfy the statutory requirement for “substantial evidence of effectiveness.” In response, PTC elected to pull the application rather than continue the review process.

The company says it is “disappointed that FDA approval cannot be achieved” after more than two decades of development work focused on patients with nonsense mutation DMD in the U.S.

FDA accepted the resubmission for review 10/2024. In 2017, the agency issued a complete response letter on the original NDA, which called for effectiveness evidence from an additional adequate and well-controlled clinical trial. The letter also mentioned other nonclinical and manufacturing matters that PTC said it would address at the time.

The company said the resubmission was based on data from a placebo-controlled trial (Study 041). “Following 72 weeks of Translarna treatment, there was significant benefit demonstrated on the key study endpoints of six-minute walk distance (6MWD) (p=0.0248), NorthStar Ambulatory Assessment (p=0.0283), 10-meter walk/run (p=0.0422), four-stair climb (p=0.0293), and time to 10% worsening of 6MWD (p=0.0078),” the company said.

Additionally, PTC said the filing included the findings on long-term Translarna treatment benefit as documented in the STRIDE registry. “Translarna treatment resulted in a 3.5-year delay in loss of ambulation (p<0.0001) and a 1.8-year delay in reaching a predicted forced vital capacity of less than 60% (p=0.0028) a critical threshold of lung function,” it added.

Translarna is described as a protein restoration therapy designed to enable the formation of a functioning protein in patients with genetic disorders caused by a nonsense mutation, which is an alteration in the genetic code that prematurely halts the synthesis of an essential protein. “The resulting disorder is determined by which protein cannot be expressed in its entirety and is no longer functional, such as dystrophin in Duchenne,” the company says.

PTC did not disclose whether it plans additional studies or regulatory interactions in the U.S. Translarna has faced a protracted regulatory path in the U.S., with prior FDA reviews also raising questions about the robustness of clinical evidence.

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