RARE Daily

Health Canada Approves Ultragenyx’ Drug for Group of Rare Metabolic Disorders

February 17, 2021

Rare Daily Staff

Health Canada has approved Ultragenyx Pharmaceutical’s Dojolvi as a source of calories and fatty acids for the treatment of patients with a set of rare metabolic conditions known as long-chain fatty acid oxidation disorders.

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.

Ultragenyx won approval for Dojolvi in the United States in June 2020.

“The number of options available to treat patients with rare long chain fatty oxidation defects have been quite limited for many years,” said Pranesh Chakraborty, associate professor, Department of Pediatrics, University of Ottawa, executive director of Newborn Screening Ontario, and co-lead of the Canadian Inherited Metabolic Diseases Research Network. “Clinicians and patients, who have been working with researchers over many years to study the safety and efficacy of Dojolvi, are very excited to have this new therapy available to help patients in Canada.”

Ultragenyx said it expects Dojolvi to be available to patients in Canada in April and will be working closely with private insurers and through the public drug plan process to pursue coverage for Dojolvi as quickly as possible.  

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