Drugs

Drug for Ultra-Rare Mitochondrial Disease Approved

FDA has approved UCB’s Kygevvi (doxecitine and doxribtimine), which the company describes as the first treatment for thymidine kinase 2 deficiency (TK2d), a life-threatening genetic mitochondrial disease characterized by progressive muscle weakness. The approval covers adults and pediatric patients whose symptoms begin at or before 12 years of age.

TK2d is an ultra-rare condition, affecting an estimated 1.6 people per million, and can be fatal within three years of symptom onset in children. Until now, patients had no treatment options beyond supportive care.

Approval is based on data from a Phase 2 study, two retrospective chart reviews, and an expanded access program, including 82 patients with symptom onset before age 12, according to the company. Compared with an external control group, Kygevvi reduced the overall risk of death from the start of treatment by about 86% (95% CI: 61–96%). Median treatment duration was four years, and median age of symptom onset was 1.5 years, it says.

The company says common adverse reactions include diarrhea, vomiting, abdominal pain, and elevated liver enzymes.

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