NOVATO, Calif., Aug. 23, 2021 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases, today announced that Brazil’s National Health Surveillance Agency (ANVISA) 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).
NOVATO, Calif., Feb. 17, 2021 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultra-rare genetic diseases, today announced that Health Canada has approved Dojolvi™ (triheptanoin) as a source of calories and fatty acids for the treatment of adult and pediatric patients with long-chain fatty acid oxidation disorders (LC-FAOD). Long-chain fatty acid oxidation disorders (LC-FAOD) are a group of rare, genetic, life-threatening disorders caused by defects in the enzymes needed to produce energy from fatty acids. Dojolvi is a highly purified, synthetic, 7-carbon fatty acid triglyceride specifically designed to provide medium-chain, odd-carbon fatty acids as an energy source and metabolite replacement for people with LC-FAOD.
NOVATO, Calif., July 22, 2020 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for rare and ultra-rare diseases, today announced that Dojolvi™ (triheptanoin) is now commercially available. The U.S. Food and Drug Administration (FDA) approved Dojolvi on June 30, 2020 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), a group of rare, lifelong, and life-threatening genetic disorders in which the body is unable to convert long-chain fatty acids into energy.
The FDA has approved a second rare disease drug from California-based Ultragenyx in the space of a fortnight, for treatment of long-chain fatty acid oxidation disorders.
PITTSBURGH, July 2, 2020 /PRNewswire/ -- PANTHERx® Rare Pharmacy announces that it has been selected by Ultragenyx Pharmaceutical as a limited distribution partner for new drug DOJOLVI™ (triheptanoin). DOJOLVI™ (a highly purified, pharmaceutical-grade, synthetic seven carbon fatty acid triglyceride) is a specially designed synthetic triglyceride compound, which was developed to treat patients with long-chain fatty acid oxidation disorders (LC-FAOD). DOJOLVI™ provides these patients with a medium-length, odd-chain fatty acid that LC-FAOD patients can metabolize and serve as an alternative source of energy (by increasing intermediate substrates in the Krebs cycle, a key energy-generating process).
Zogenix’s Fintepla (fenfluramine) has been approved for the treatment of seizures associated with Dravet syndrome in patients aged 2 years or older. The syndrome is a rare, life-threatening form of epilepsy.
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for rare and ultra-rare diseases, today announced that the U.S. Food and Drug Administration (FDA) 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). LC-FAOD 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. Dojolvi is a highly purified, synthetic, 7-carbon fatty acid triglyceride specifically designed to provide medium-chain, odd-carbon fatty acids as an energy source and metabolite replacement for people with LC-FAOD.
The FDA accepts for review Ultragenyx's (NASDAQ:RARE) marketing application seeking approval of UX007 (triheptanoin) for the treatment of long-chain fatty acid oxidation disorders (LC-FAOD), a group of inherited diseases in which the body is unable to convert long-chain fatty acids into energy.
Ultragenyx Pharmaceutical (RARE) neared a breakout Wednesday after the Food and Drug Administration said it would consider approving the biotech's latest drug ahead of the usual schedule.