Drugs

FDA Accepts NDA for Menkes Disease Drug

FDA has accepted for review a Fortress Biotech NDA for CUTX-101 (copper histidinate) for treating Menkes disease, a rare X-linked recessive pediatric disease caused by gene mutations of the copper transporter ATP7A. The submission has been granted priority review and a user fee action date of 6/30.

Menkes disease, which primarily affects male infants, leads to copper accumulating at abnormally low levels in the liver and brain, but at higher-than-normal levels in the kidney and intestinal lining.

The NDA is based on clinical data showing statistically significant improvement in overall survival for Menkes disease subjects who received early treatment with CUTX-101, with a nearly 80% reduction in death risk compared to an untreated historical control cohort, the company says. “Median overall survival was 177.1 months for CUTX-101 early treatment cohort compared to 16.1 months for the untreated historical control cohort,” it says. CUTX-101 was previously granted FDA Breakthrough Therapy, Fast Track, Rare Pediatric Disease and Orphan Drug designations.

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