Drugs

BioMarin Hold Extended as FDA Seeks More Data

FDA has extended a clinical hold on BioMarin Pharmaceutical’s PHEARLESS Phase 1/2 study of BMN 307, an AAV5-human phenylalanine hydroxylase gene therapy being studied in adults with phenylketonuria. The trial has been on hold since 9/2021, and now FDA has requested data from additional non-clinical studies to assess the theoretical oncogenic risk to human study participants, which is expected to take several quarters, according to the company.

At the time of the initial hold, a company statement said the clinical hold was based on interim safety findings from a pre-clinical, non-GLP (good laboratory practice) pharmacology study. It further said that BioMarin was pausing further enrollment in the global Phase 1/2 study pending investigation of findings of cancers in some rodents treated with the drug.

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