Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE)

Ultragenyx Announces FDA Accepts Proposal to Submit an NDA for UX007 for the Treatment of Long-Chain Fatty Acid Oxidation Disorders
GlobeNewswire•August 29, 2018
NOVATO, Calif., Aug. 29, 2018 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has accepted Ultragenyx’s most recent proposal to submit a New Drug Application (NDA) for UX007 for the treatment of long-chain fatty acid oxidation disorders (LC-FAOD) based on existing data. Further details regarding timing will be forthcoming following a pre-NDA meeting, which is being scheduled for the second half of 2018.

“We appreciate FDA’s review of multiple data submissions and collaboration with us to develop a path for an early filing, and it is our commitment to get this important potential treatment to patients with this serious disease as quickly as possible,” said Emil D. Kakkis, M.D., Ph.D., Chief Executive Officer and President of Ultragenyx. “We will meet with the FDA to discuss the details of the NDA submission and, if approved, appropriate post-approval commitments to further evaluate long-term outcomes of UX007 in patients with LC-FAOD.”

The data submitted to the FDA for evaluation included the recently published 78-week Phase 2 study results in 29 patients, a now published retrospective medical record review of 20 patients, and 56 emergency IND cardiomyopathy and other patients. 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 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.

In the EU, Ultragenyx will discuss these data with the European Medicines Agency (EMA) and expects to have an update in the second half of 2018.