Drugs

CDER Denies PTC Therapeutics Appeal on Duchenne Drug

CDER’s Office of Drug Evaluation 1 has denied a PTC Therapeutics appeal of a 2/22 refuse-to-file letter issued by the Division of Neurological Products on an NDA for Translarna (ataluren) for treating nonsense mutation Duchenne muscular dystrophy (nmDMD). At the time, the agency said the submission was not sufficiently complete to permit a substantive review, according to the company.

PTC intends to escalate its appeal to the next supervisory level at 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,” it says. 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 agency. “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,” it says. “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.”

Primarily affecting males, DMD is a progressive muscle disorder caused by the lack of functional dystrophin protein. Patients with DMD lose the ability to walk as early as age 10 and experience life-threatening lung and heart complications in their late teens and twenties. Nonsense mutations account for 13% of DMD cases, or about 2,000 patients in the U.S., according to PTC.

Gupta Raises Concerns About Overton’s Independence, Ideology

FDA commissioner nominee Heidi Overton’s responses on vaccines, contraception and abortion medication during her 9/24 Senate confirmation hearing raise concerns that ideology could influence her decision-making if she takes over the agency, MSNBC senior medical analyst Dr. Vin Gupta said Friday. Gupta was particularly critical of Overton’

Overton Confirmation Remains Uncertain After Senate Hearing

FDA commissioner nominee Heidi Overton emerged from her Senate confirmation hearing Thursday (see earlier story) without resolving questions about whether she has enough Republican support to advance — and with a rapidly narrowing window for confirmation before the midterm elections. The Senate Health, Education, Labor and Pensions Committee hearing provided few

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA has classified Boston Scientific's recall of its Imager II Angiographic Catheters as Class I because a manufacturing problem could cause catheter tips to degrade and detach, potentially resulting in life-threatening complications. The agency announced the Class I classification 9/25, updating an Early Alert it issued

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome

FDA has approved Roche’s Gazyva (obinutuzumab) for reducing the risk of relapse in adults and children aged 2 and older with frequently relapsing or steroid-dependent, childhood-onset idiopathic nephrotic syndrome who are in remission. The expanded approval covers the use of the CD20-directed antibody beyond its existing

Read more

Gupta Raises Concerns About Overton’s Independence, Ideology

Overton Confirmation Remains Uncertain After Senate Hearing

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome