FDA accepts proposal for NDA for UX 007 for the treatment of long-chain fatty acid oxidation disorders.- Ultragenyx Pharmaceutical Inc.

Ultragenyx Pharmaceutical Inc. announced that the FDA has accepted Ultragenyx�s most recent proposal to submit a New Drug Application (NDA) for UX 007 (triheptanoin) 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.

The data submitted to the FDA for evaluation included the recently published 78-week Phase II 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 II 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 UX 007 compared to an annualized rate of MCEs in the 18 months prior to treatment with UX 007. 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 UX 007 treatment. The safety profile was consistent with what has been previously observed with UX 007.

< Comment: Triheptanoin is intended as a substrate replacement therapy to restore deficient intermediates in the mitochondria and to enable energy metabolism in patients with FAOD and potentially other genetic metabolic disorders. The company licensed exclusive North American rights to triheptanoin from Baylor Research Institute and has an exclusive option to an exclusive license for the rest of world.