Drugs

FDA Priority Review for Regeneron Pozelimab

FDA has accepted for priority review a Regeneron BLA for pozelimab (C5 antibody) to treat children and adults with CHAPLE disease. A company statement says CHAPLE is an ultra-rare disease also known as CD55 deficiency with Hyperactivation of complement, Angiopathic thrombosis and Protein Losing Enteropathy or CD55-deficient protein-losing enteropathy. There currently are no approved treatments for CHAPLE disease, it says. The PDUFA target action date on the BLA is 8/20.

Pozelimab is described as an investigational fully human monoclonal antibody designed to block the activity of complement factor C5, a protein involved in complement system activation.

The BLA is supported by results from a Phase 2/3 open-label trial that investigated its safety and efficacy in 10 patients over 12 months old. Regeneron says that at 24 weeks, the co-primary endpoints were achieved with 100% of patients experiencing rapid and sustained normalization of serum albumin (a disease biomarker) and improvement or no worsening of clinical symptoms.

FDA designated pozelimab for treating CHAPLE as a drug for a “rare pediatric disease” in 4/2020 with the opportunity for Regeneron to receive a rare pediatric disease priority review voucher if pozelimab is approved for CHAPLE. The drug was granted an orphan drug designation at the same time. It was granted fast track designation in 9/2022.

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