Drugs

Translarna Effectiveness ‘Not Persuasive’: FDA

FDA medical reviewers say that the studies submitted by PTC Therapeutics supporting its NDA for Translarna (ataluren) to treat dystrophinopathies [most notably Duchenne muscular dystrophy (DMD)] resulting from nonsense mutations in the dystrophin gene (nmDMD) do not persuasively demonstrate the drug’s effectiveness. The agency’s Peripheral and Central Nervous System Drugs Advisory Committee is being asked to discuss the studies’ interpretability and persuasiveness at its 9/28 meeting.

The reviewers’ briefing document distributed in advance of the meeting notes that the advisory committee “is in the unusual position of being asked to provide input on an application that has been filed-over-protest.” It says FDA issued refuse-to-file applications for ataluren for treating nmDMD in 2011 and 2016. The company appealed each refusal and the appeals were denied. FDA says the sponsor “has chosen to insist that this most recent application be filed over protest.”

According to the reviewers, the application “contains a large number of exploratory analyses that lack interpretability and are often entirely based on unblinded data. The presentation of the data in the application is often unclear as to which analyses were used by the applicant. Ultimately, no positive results from any prospectively planned analyses that are persuasive have been provided with this application.”

Observers say the agency will be under close scrutiny as it evaluates this application because last year CDER director Janet Woodcock approved Sarepta Therapeutics’ DMD drug Exondys 51 (eteplirsen) against the recommendations of agency staff and an advisory committee. The reviewers appealed the approval to then-FDA commissioner Robert Califf, who sided with Woodcock.

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