RARE Daily

Ultragenyx Wins FDA Approval for Treatment for Patients with Group of Rare Metabolic Disorders

July 1, 2020

The U.S. Food and Drug Administration approved Ultragenyx Pharmaceutical’s Dojolvi as a source of calories and fatty acids for the treatment for patients with a set of rare metabolic disorders.

Photo: Camille Bedrosian, chief medical officer of Ultragenyx

The approval for Dojolvi is for pediatric and adult patients with molecularly confirmed long-chain fatty acid oxidation disorders (LC-FAOD).

LC-FAOD are a group of autosomal recessive genetic disorders characterized by metabolic deficiencies in which the body is unable to convert long-chain fatty acids into energy. The inability to produce energy from fat can lead to severe depletion of glucose in the body and serious complications, which can lead to hospitalizations or early death. LC-FAOD are included in newborn screening panels across the United States and in certain European countries due to the risk for serious outcomes including death early in life. Other current treatment options for LC-FAOD include avoidance of fasting, low-fat/high-carbohydrate diets, carnitine and even-carbon medium-chain triglyceride (MCT) oil, a medical food product.

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.

“With today’s FDA approval, patients living with this serious, unpredictable, and often catastrophic disease now have an approved therapy,” said Camille Bedrosian, chief medical officer of Ultragenyx. “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.”

Ultragenyx expects Dojolvi to be available to patients in the next 30 days.

Author: Rare Daily Staff

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