Drugs

Ultragenyx Gets FDA Nod to File UX007 NDA

FDA has accepted an Ultragenyx Pharmaceutical proposal to submit an NDA for UX007 for treating long-chain fatty acid oxidation disorders based on existing data. A pre-NDA meeting is being scheduled for later this year to iron out the submission details, the company says.

Data submitted to FDA for evaluation included data from a recently published 78-week Phase 2 study in 29 patients, a retrospective medical record review of 20 patients, and 56 emergency IND cardiomyopathy patients, Ultragenyx says. In addition, these data were supported by results of a published randomized controlled investigator study of 32 patients showing an effect of triheptanoin on cardiac function. “In the 78-week sponsored Phase 2 study, the data showed a 48.1% reduction in the mean annualized rate of major clinical events (MCEs) and a 50.6% reduction in the median annualized rate of MCEs after 78 weeks of treatment with UX007 compared to an annualized rate of MCEs in the 18 months prior to treatment with UX007,” it says.

Ultragenyx says UX007 is a highly purified, pharmaceutical-grade, synthetic, seven-carbon fatty acid triglyceride created via a multi-step chemical process. “Unlike typical even-chain fatty acids, UX007 can be converted to new glucose through the Krebs cycle, potentially providing an important added therapeutic effect, particularly when glucose levels are too low,” it says.

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