Ultragenyx announces Canadian approval of Dojolvi to treat long-chain fatty acid oxidation disorders in adults and children.

Ultragenyx Pharmaceutical Inc 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. 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 marked 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 Canada due to the risk of 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. LC-FAOD affects an estimated 8,000 to 14,000 children and adults in the developed world, including Canada. “The number of options available to treat patients with rare long chain fatty oxidation defects have been quite limited for many years,” said Dr. 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 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. To support access, Ultragenyx’s UltraCare program helps patients and caregivers understand insurance coverage. The UltraCare program may also assist patients in finding support for access to Ultragenyx medicines, including Dojolvi.