Advisory Committee Meeting Scheduled for Translarna

PTC Therapeutics announced today that the U.S. Food and Drug Administration (FDA) has tentatively scheduled a Peripheral and Central Nervous Systems Drugs Advisory Committee meeting for Sept. 28, 2017, to review the new drug application for Translarna to treat some forms of Duchenne muscular dystrophy (DMD).

Part of the FDA mission is to evaluate new therapies and determine which are safe and effective for their intended use. Advisory committees provide the FDA with independent advice from outside experts on issues related to human and veterinary drugs, vaccines, medical devices and food.

The Peripheral and Central Nervous System Drugs (PCNS) Advisory Committee reviews and evaluates data concerning the safety and effectiveness of marketed and investigational human drug products for use in the treatment of neurologic diseases and makes appropriate recommendations to the Commissioner of Food and Drugs. One of several dozen technical and scientific advisory committees, it is responsible for reviewing potential treatments for DMD.

FDA to make a decision on Translarna by Oct. 24

PTC completed submission of its New Drug Application (NDA) to market Translarna in the U.S. in January 2016, but in February received a Refuse to File letter from the FDA stating that the application was not sufficient to permit a substantive review. It submitted an appeal in July to the next level of FDA management and reported in October that the appeal had been denied.

PTC used the FDA’s “file over protest” regulations to file the NDA. These regulations allow a company to have its NDA filed and reviewed following a refuse to file determination.

Translarna received “conditional approval” in the European Union in August 2014 for use in people with DMD caused by a nonsense mutation who are at least 5 years old and able to walk.

An FDA decision on the drug is expected on or before Oct. 24, 2017.

Translarna may slow functional decline

Translarna is under development by PTC to treat DMD caused by a type of genetic mutation known as a “nonsense mutation” or “premature stop codon.” This type of mutation results in production of a protein that is not complete and therefore not functional.

A “read-through” drug, Translarna is designed to act by changing the way muscle cells interpret genetic information, coaxing them to produce a needed muscle protein called dystrophin despite the presence of a nonsense mutation in the dystrophin gene. The drug, which is taken by mouth, may help approximately 13 percent of people with DMD.

In October 2015, PTC announced results from its phase 3 “ACT DMD” clinical trial of Translarna, which demonstrated that the drug failed to meet its primary endpoint. However, a post-hoc analysis indicated that the drug may slow functional decline in a subset of DMD patients, while other patients were not responsive to treatment. Among those who responded, treatment was associated with statistically significant improvements to several clinical outcome measures.

MDA has contributed nearly $3 million towards Translarna’s development and phase 2 clinical testing.

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