Drugs

Boxed Warning for Adzynma Enzyme Replacement

FDA says it has directed Takeda to add a Boxed Warning to the labeling for its Adzynma, a recombinant ADAMTS13 enzyme replacement therapy approved for congenital thrombotic thrombocytopenic purpura (cTTP). An agency online notice says Adzynma has been associated with the development of neutralizing antibodies that can prevent the drug from working in serious cases. It says this has resulted in death, including that of a seven-year-old child.

Adzynma is described as a lab-manufactured enzyme replacement therapy, which FDA approved on 11/9/2023, for both routine prevention and on-demand treatment of cTTP in adults and pediatric patients. Adzynma is the only FDA-approved recombinant ADAMTS13 therapy for cTTP, a rare, inherited blood disorder caused by a severe deficiency of the ADAMTS13 enzyme, which is essential for preventing abnormal blood clotting. The drug is said to contain two slightly modified forms of the ADAMTS13 protein, one of which matches the natural human sequence.

The notice says a safety signal associated with Adzynma was identified through the FDA Adverse Event Reporting System.

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