FDA accepts new drug application for UX 007 for treatment of long-chain fatty acid oxidation disorders.- Ultragenyx Pharmaceutical
Ultragenyx Pharmaceutical Inc.announced that the FDA has accepted for review the company�s New Drug Application (NDA) for UX 007 (triheptanoin) for the treatment of long-chain fatty acid oxidation disorders (LC-FAOD), a group of genetic disorders in which the body is unable to convert long-chain fatty acids into energy.
The FDA has assigned a standard review designation with a Prescription Drug User Fee Act (PDUFA) target date of July 31, 2020.
The FDA has indicated that it is not currently planning to hold an advisory committee meeting to discuss the application. The NDA filing is supported by a comprehensive package of data including results from a company-sponsored Phase II study of UX 007 in 29 patients, a long-term safety and efficacy extension study in 75 patients including 20 patients who were previously na�ve to UX 007, a retrospective medical record review of 20 original compassionate use patients, 67 patients treated through expanded access, and a randomized controlled investigator-sponsored study of 32 patients showing an effect on cardiac function.
Learning Zones
The Learning Zones are an educational resource for healthcare professionals that provide medical information on the epidemiology, pathophysiology and burden of disease, as well as diagnostic techniques and treatment regimens.
2 mins