FDA Policy/General

Ultragenyx Pharmaceutical Plans NDA Mid-2019

Ultragenyx Pharmaceutical has completed a pre-NDA meeting and plans to submit an NDA for UX007 for treating patients with long-chain fatty acid oxidation disorders in mid-2019. The company says the meeting led to an agreement to include data from a Phase 2 company-sponsored study of UX007 in 29 patients, a long-term efficacy and safety extension study in 75 patients, a retrospective medical record review of 20 patients, data from 56 patients treated through expanded access, and a randomized controlled investigator-sponsored study of 32 patients showing an effect of triheptanoin on cardiac function.

In the Phase 2 study, the data showed a 48.1% reduction in the mean annualized rate of major clinical events and a 50.6% reduction in the median annualized rate of major events after 78 weeks of treatment with UX007, the company says. The pre-NDA meeting also clarified the analyses and data presentations the agency will look for in the submission, it says.

Ultragenyx says UX007 is a highly purified, pharmaceutical-grade, synthetic, seven-carbon fatty acid triglyceride created via a multi-step chemical process. It is designed to provide patients with “medium-length, odd-chain fatty acids that can be metabolized to increase intermediate substrates in the Krebs cycle, a key energy-generating process,” it says. “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.”

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

Read more

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