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Tuesday, June 30, 2020

FDA OKs Ultragenyx’s Dojolvi for rare metabolic disorders

The FDA approves Ultragenyx’s (NASDAQ:RARE) Dojolvi (triheptanoin) for the treatment of pediatric and adult patients with molecularly confirmed long-chain fatty acid oxidation disorders (LC-FAOD), a group of rare inherited disorders in which the body is unable to synthesize long-chain fatty acids into energy.
Triheptanoin 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 company says Dojolvi should be available in 30 days.
Management is currently hosting a conference call to discuss the nod.

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