Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for rare and ultra-rare diseases announced that the U.S. Food and Drug Administration has approved Dojolvi (triheptanoin) as a source of calories and fatty acids for the treatment of pediatric and adult patients with molecularly confirmed long-chain fatty acid oxidation disorders (LC-FAOD). They are a group of rare, lifelong and life-threatening genetic disorders in which the body is unable to convert long-chain fatty acids into energy.
Ultragenyx expects Dojolvi to be available to patients in the next 30 days. To support access, Ultragenyx’s UltraCare program helps patients and caregivers understand insurance coverage and assists them in finding financial support for Ultragenyx medicines, including Dojolvi, and for the administration of them. Dedicated in-house UltraCare Guides are available Monday through Friday from 9 a.m. to 8 p.m. Eastern Time at 888-756-8657.