PTC Therapeutics (PTCT) Says FDA Denied First Appeal of the Refuse to File Letter

October 17, 2016 8:02 AM EDT

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PTC Therapeutics, Inc. (NASDAQ: PTCT) today provided a regulatory update on Translarna™ (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD).

U.S. regulatory update

PTC Therapeutics announced today that at the end of last week, the Office of Drug Evaluation I (ODE-I) of the U.S. Food and Drug Administration (FDA) denied the company's first appeal of the refuse to file letter issued by the FDA's Division of Neurological Products (DNP) on February 22, 2016 regarding PTC's New Drug Application (NDA) for Translarna for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD).

The company intends to escalate its appeal to the next supervisory level of the FDA. This is an iterative process and the company anticipates that multiple cycles of appeals to progressively higher levels of the FDA may be required.

PTC continues to assert that a proper assessment of the data and analyses from multiple clinical studies, including two of the largest placebo-controlled trials ever conducted in DMD, can only be accomplished in the context of a full and fair review by the FDA. This would include an advisory committee meeting that allows clinical experts and representatives of the patient community to express their views on Translarna for the treatment of nmDMD. The company believes that Translarna is the only therapy in clinical development designed to target the underlying cause of nmDMD. In addition, a favorable safety profile has been consistently demonstrated in PTC's clinical trials, which have enrolled over 1,000 individuals to date.

"We believe that fair consideration of the totality of Translarna's data requires a full review of our application by the FDA," said Stuart W. Peltz, Ph.D., Chief Executive Officer, PTC Therapeutics, Inc. "In light of this, continuing the formal dispute resolution process reflects our ongoing commitment to work with regulators and the Duchenne community to make Translarna available to nmDMD patients in the United States."

In addition, the company maintains its position that PTC should, under existing law and in fairness to patients, be provided the same opportunity for full review that the DNP gave to other recent applicants for products in development for different subsets of the DMD population.

"I am disappointed that a treatment for patients with nonsense mutation DMD is still not receiving a fair opportunity in front of the FDA," said Pat Furlong, President and Founder of Parent Project Muscular Dystrophy. "This inconsistency is unacceptable and is concerning for the entire community. This devastating, muscle-wasting disease cuts short the lives of boys and young men and every day that we wait for treatments, is a day in which muscle function is lost and not regained. As a community, we cannot rest until there are treatments for all the boys and young men."

European regulatory update

PTC recently participated in an oral explanation meeting before the European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) in connection with the company's ongoing annual renewal of its marketing authorization for Translarna for the treatment of nmDMD in ambulatory patients aged five years and older. The marketing authorization in the European Economic Area (EEA), originally granted in August 2014, is subject to an annual renewal process, including an assessment by European regulators of a risk-benefit profile in favor of Translarna authorization.

Following conclusion of the recent oral explanation, the CHMP issued a request for supplemental information (RSI), including a request categorized as a major objection. Generally speaking, renewal of a marketing authorization requires a company to adequately address the points raised in a major objection. As with prior RSIs received by the company during this renewal process, the major objection relates to the efficacy and overall risk-benefit profile of Translarna as well as the design and conduct of an additional clinical trial that would provide comprehensive clinical data. The RSIs also include requests categorized as other concerns, which do not rise to the level of a major objection, and are generally associated with the primary pharmacology of Translarna and label matters.

The company continues to believe that if the CHMP issues a positive opinion in favor of the renewal of Translarna's marketing authorization, such renewal, and any subsequent annual renewals, will be coupled with an obligation to conduct an agreed upon new clinical trial of Translarna for the treatment of nmDMD. The EMA could also impose other new conditions to the authorization for renewal or make other recommendations, including the potential withdrawal of the marketing authorization.

PTC anticipates that an opinion regarding its marketing authorization renewal request will be adopted by the CHMP before the end of 2016. The company expects that its current marketing authorization will remain valid while the EMA's assessment is ongoing and until it is concluded with a decision from the European Commission.



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