FDA rejects PTC Therapeutics’ application for Duchenne drug

The drug regulator said that an additional clinical trial would be required to establish the efficacy of the protein restoration therapy ataluren for the rare and fatal genetic disorder that mainly affects young boys.

FDA’s decision on ataluren was on expected lines after an advisory panel voted 10-1 last month against recommending approval of the drug, citing that it did not work in two major patient tests while calling the provided data to be inconclusive.

PTC Therapeutics said it strongly disagrees with the FDA ruling on ataluren and plans to file a formal dispute resolution request.

PTC Therapeutics CEO Stuart Peltz said: “We are extremely disappointed for the Duchenne community and strongly disagree with the agency's conclusions.

“We believe that this decision fails to consider the benefit-risk of ataluren and the high unmet medical need.”

A complete response letter (CRL) from the Office of Drug Evaluation I of the drug regulator stated that it could not approve PTC’s application for ataluren in its current form.

The CRL particularly asks PTC Therapeutics to come up with an additional adequate and well-controlled clinical trial(s) to prove the effectiveness of ataluren.

PTC Therapeutics revealed that it is in the process of addressing other nonclinical and CMC matters mentioned by the FDA in the letter.

Ataluren, which is traded under the name Translarna, has a conditional approval in Europe, where its license needs to be renewed every year. The approval is for the treatment of nmDMD in ambulatory patients aged five years and older.

Image: FDA Building 51 houses the Center for Drug Evaluation and Research. Photo: courtesy of The U.S. Food and Drug Administration.