Benzinga | Aug 23, 2021 08:32AM EDT

Ultragenyx Pharma Reports Approval Of Dojolvi In Brazil For Treatment Of Long-Chain Fatty Acid Oxidation Disorders In Adults, Children

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(r) (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.