Drugs

Duchenne Drug Reviewer Farkas Leaves FDA for Parexel

Investors and Duchenne muscular dystrophy patients and their families are cheering the departure of CDER Division of Neurology Products clinical team leader Ronald Farkas, who joined clinical research organization Parexel 9/9. Farkas was one of the harshest critics of Sarepta’s NDA for Duchenne drug eteplirsen during an April advisory committee meeting that voted 7 to 6 (see story) that the company did not provide substantial evidence from adequate and well controlled studies that the drug induces production of dystrophin to a level that is reasonably likely to predict clinical benefit in patients. It didn’t take Wall Street long to react to Farkas’ subsequent departure – Sarepta stock was up about 25% in mid-day trading Wednesday.

In June, 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 said at the time that it planned to submit data from 13 patient biopsy samples, at baseline and week 48, to the agency within a 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. In May, FDA extended the user fee review goal to continue to sort through several data issues.

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