Posted by Michael Wonder on 17 Apr 2019
Ultragenyx announces UX007 granted fast track designation and rare pediatric disease designation by U.S. FDA for treatment of long-chain fatty acid oxidation disorders
16 April 2019 - Company on track to submit NDA to FDA in mid 2019.
Ultragenyx Pharmaceutical today announced that the U.S. FDA has granted fast track designation and rare pediatric disease designation to UX007 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.
Ultragenyx is on track to submit a new drug application in mid-2019. The submission will include data from a company-sponsored Phase 2 study of UX007 in 29 patients, data from a long-term safety and efficacy extension study in 75 patients, a retrospective medical record review of 20 original compassionate use patients, data from 70 patients treated through expanded access, and a randomized controlled investigator-sponsored study of 32 patients showing an effect of triheptanoin on cardiac function.
