Drugs

FDA Asks for More Data on Duchenne Drug NDA

Sarepta Therapeutics and its eteplirsen for treating Duchenne muscular dystrophy received a shot in the arm 6/6 when, in an apparent compromise with FDA, the company agreed to an agency request that it provide dystrophin data, as measured by western blot, from biopsies already obtained from an ongoing confirmatory study of eteplirsen (PROMOVI), as part of its ongoing NDA evaluation. The company plans to submit data from 13 patient biopsy samples, at baseline and week 48, to the agency over the next few weeks. In a statement, Sarepta said it expects this will “facilitate a prompt decision on the NDA by the agency.” Eteplirsen is designed to address the underlying cause of Duchenne’s by restoring the dystrophin messenger RNA (mRNA) reading frame, and enabling the production of a shorter, functional form of the dystrophin protein, according to the company.

Last month, FDA extended its NDA review, and the submission has become a politically charged issue, with U.S. lawmakers weighing in and urging the agency to exercise its flexibilities when making a review decision. At the time, the agency reportedly communicated that it will continue to work past the 5/25 user fee goal date and strive to complete its work in as timely a manner as possible.

In April, FDA’s Peripheral and Central Nervous System Drugs Advisory Committee voted 7 to 6 (see story) that a Sarepta Therapeutics NDA did not provide substantial evidence from adequate and well controlled studies that eteplirsen induces production of dystrophin to a level that is reasonably likely to predict clinical benefit in Duchenne patients. The vote was seen as a setback for the company’s bid to gain accelerated approval for the drug. A separate 7 to 3 vote by the panel recommended that data were not sufficient to meet traditional approval standards. However, before those votes, CDER director Janet Woodcock obliquely hinted at a more positive assessment of the drug in an unusual appearance she made before the panel.

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

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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