Drugs

X4 Pharm NDA for WHIM Syndrome

X4 Pharmaceuticals has submitted an NDA for a once-daily, oral mavorixafor to treat individuals aged 12 and older with WHIM (warts, hypogammaglobulinemia, infections, and myelokathexis) syndrome, a rare immunodeficiency. The submission is supported by data from the pivotal 4WHIM Phase 3 clinical trial in individuals with WHIM syndrome. The trial met its primary endpoint and key secondary endpoint, with no treatment-related serious adverse events reported and no discontinuations due to safety concerns, the company says.

X4 says the trial data also revealed that mavorixafor treatment “resulted in reductions in the rate, severity, and duration of infections in trial participants versus placebo.” The data were published in oral presentations at the annual meetings of both the Clinical Immunology Society and European Hematology Association.

Mavorixafor is an investigational small-molecule antagonist of CXCR4, X4 says. It previously has been granted a Breakthrough Therapy Designation, Fast Track Designation, Rare Pediatric Designation, and orphan drug status.

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