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Ultragenyx Announces Approval of Dojolvi® (triheptanoin) in Brazil for the Treatment of Long-chain Fatty Acid Oxidation Disorders in Adults and Children
Dojolvi is the only approved therapy for this rare, life-threatening metabolic disorder NOVATO, Calif., Aug. 23, 2021 (GLOBE NEWSWIRE) -- Ultragenyx
About this update from Ultragenyx Pharmaceutical Inc.
[{"type":"text","content":"Dojolvi is the only approved therapy for this rare, life-threatening metabolic disorder\nNOVATO, Calif., Aug. 23, 2021 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases, today announced that Brazil’s National Health Surveillance Agency (ANVISA) has approved Dojolvi® (triheptanoin) as a source of calories and fatty acids for the treatment of pediatric and adult patients with molecularly confirmed long-chain fatty acid oxidation disorders (LC-FAOD). “Patients with LC-FAOD face frequent hospitalizations and major medical events with limited options to support management of their disease,” said Eduardo Thompson, Senior Vice President and Regional Head, Latin America at Ultragenyx. “This approval of Dojolvi is critical to providing therapeutic options for patients in Brazil and we are now working with urgency to obtain reimbursement approval in order to support broad access to therapy. We are grateful to the community of patients, caregivers and physicians whose efforts have contributed to this important approval.” Dojolvi is also approved by the U.S. Food and Drug Administration (FDA) and by Health Canada for the treatment of adult and pediatric patients with LC-FAOD. Outside of the U.S., patients have had access to Dojolvi through named patient and early access programs. About LC-FAODLC-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. 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 affect an estimated 8,000 to 14,000 children and adults in the developed world and are included in newborn screening panels in various geographies due to the risk for serious outcomes including death early in life. The Brazilian government recently sanctioned law 14.154/2021 that expands the list of diseases to be included in the National Newborn Screening Program carried out i...