Ultragenyx Announces Intent to Submit New Drug Application to U.S. FDA for UX007

Ultragenyx Pharmaceutical (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases, today announced that it has completed a pre-NDA meeting with the U.S. Food and Drug Administration (FDA) and plans to submit a New Drug Application (NDA) to the FDA for UX007 for the treatment of patients with long-chain fatty acid oxidation disorders (LC-FAOD) in mid-2019.

As quoted in the press release:

“In our recent pre-NDA meeting with the FDA, we were pleased to reach an agreement on the details of our regulatory submission,” said Camille L. Bedrosian, M.D., Chief Medical Officer of Ultragenyx. “We look forward to working with the FDA throughout the review process, and it is our commitment to bring this potential treatment to patients with this serious disease as quickly as possible.”

The NDA package will include data from the 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 78-week sponsored Phase 2 study, the data showed a 48.1 percent reduction in the mean annualized rate of major clinical events (MCEs) and a 50.6 percent 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. There was also a 50.3 percent reduction in the mean annualized duration of MCEs and a 76.7 percent reduction in the median annualized duration of MCEs following 78 weeks of UX007 treatment. The safety profile was consistent with what has been previously observed with UX007. The pre-NDA meeting clarified what information from these studies are considered most important by the FDA, and what analyses and data presentations they will look for in the NDA filing.  An advisory committee is under consideration for this product candidate, but no confirmation that one would be convened was received during the pre-NDA meeting.

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