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FDA approves first treatment for long-chain fatty acid oxidation disorders

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The U.S. Food and Drug Administration (FDA) has approved triheptanoin (Dojolvi) as a source of calories and fatty acids for the treatment of patients with molecularly confirmed long-chain fatty acid oxidation disorders (LC-FAOD), according to a press release from Ultragenyx Pharmaceutical Inc. People with LC-FAOD are unable to convert long-chain fatty acids into energy

Triheptanoin is a highly purified, synthetic, 7-carbon fatty acid triglyceride that is designed to deliver medium-chain, odd-carbon fatty acids to patients with LC-FAOD as an alternative energy source.

“With today’s FDA approval, patients living with this serious, unpredictable, and often catastrophic disease now have an approved therapy. Many patients with long-chain fatty acid oxidation disorders have difficult lives with frequent hospitalizations and major medical events despite the best current care. Now these patients have an approved treatment as an option to help manage their disease,” said Camille L. Bedrosian, MD, Chief Medical Officer of Ultragenyx, in the press release. “We are grateful to the patients, caregivers, families, dietitians, and doctors involved for helping to make this community milestone possible, and we are committed to ensuring that all patients in the U.S. with LC-FAOD who might benefit from Dojolvi will have access to it.”

Read the full press release here.

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