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FDA Advisory Committee: Data in Support of Translarna ‘Inconclusive’

Amy Madsen

The U.S. Food and Drug Administration (FDA) yesterday convened a meeting with the Peripheral and Central Nervous Systems Drugs Advisory Committee (PCNS), to review the new drug application, or NDA, for ataluren (brand name Translarna) to treat some forms of Duchenne muscular dystrophy (DMD).

Following a presentation of the data in support of the NDA by PTC Therapeutics and an opportunity for those on the PCNS Advisory Committee to ask questions, as well as public testimony in which individuals and family members described their experience with Translarna, members of the committee voted on the best interpretation of the information provided. The overwhelming majority (10 out of 11) suggested that although it is possible that Translarna may be effective, the data are inconclusive and more work is needed to determine whether, in fact, it is.

Ongoing trials may provide supportive data

During yesterday’s meeting, it was suggested that several studies currently being conducted by PTC could provide the additional evidence needed to confirm efficacy for Translarna.

PTC currently has three ongoing trials that may provide answers to some of the outstanding questions raised by the committee.

  • A phase 2 trial is testing Translarna in boys with DMD ages 2-5 years old.
  • A phase 3 study is testing the drug in previously treated patients with DMD.
  • A phase 3 trial is studying the long-term outcomes of Translarna in DMD.

Of the three trials, two are closed to enrollment. For information on the phase 3 long-term outcomes in DMD trial, recruiting at Seattle Children’s Hospital, contact Ana Christensen at 206-987-5433 or muscleresearch@seattlechildrens.org.

Translarna is designed to slow functional decline in DMD

Translarna is under development by PTC Therapeutics to treat DMD caused by a type of genetic mutation known as a “nonsense mutation” or “premature stop codon.” This type of mutation causes the muscle cell to stop reading the genetic code used to manufacture the muscle protein dystrophin before it reaches the end, thus preventing production of the protein.

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

FDA decision expected soon

Following the advisory committee meeting, the FDA will now complete its review and make a final decision on whether Translarna can be approved at this time.

The FDA is expected to make a decision on Translarna on or before Oct. 24.