Drugs

FDA OKs Genzyme’s Xenpozyme for ASMD

FDA has approved Genzyme’s Xenpozyme (olipudase alfa) for intravenous infusion in pediatric and adult patients with acid sphingomyelinase deficiency (ASMD). The agency says Xenpozyme is the first approved medication to treat symptoms that are not related to the central nervous system in patients with ASMD.

Xenpozyme is described as an enzyme replacement therapy that helps reduce sphingomyelinase accumulation in the liver, spleen, and lung. Its efficacy in treating ASMD was demonstrated in a placebo-controlled study of 31 patients. “Because the study had the placebo comparator and measured treatment benefits that could be detected during the study’s duration, FDA was able to conclude that Xenpozyme is effective,” the agency says. “Overall, treatment with Xenpozyme improved lung function and reduced liver and spleen size.”

The labeling has a Boxed Warning for severe hypersensitivity reactions, including anaphylaxis. Xenpozyme received fast track, breakthrough therapy, and priority review, as well as orphan drug designation. FDA awarded Genzyme a rare pediatric disease priority review voucher, an incentive to encourage the development of new drugs and biologics that prevent and treat rare diseases in children.

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