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Ultragenyx Announces U.S. Commercial Launch of Dojolvi™ (triheptanoin), the First FDA-Approved Therapy for the Treatment of Long-chain Fatty Acid Oxidation Disorders
Established UltraCare® Program Provides Ongoing Patient Support Services NOVATO, Calif., July 22, 2020 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc.
About this update from Ultragenyx Pharmaceutical Inc.
[{"type":"text","content":"Established UltraCare® Program Provides Ongoing Patient Support Services\nNOVATO, Calif., July 22, 2020 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for rare and ultra-rare diseases, today announced that Dojolvi™ (triheptanoin) is now commercially available. The U.S. Food and Drug Administration (FDA) approved Dojolvi on June 30, 2020 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), a group of rare, lifelong, and life-threatening genetic disorders in which the body is unable to convert long-chain fatty acids into energy.\n “Dojolvi, the only FDA-approved therapy for LC-FAOD, is now available for patients living with this serious, unpredictable, and often catastrophic disease,” said Erik Harris, Chief Commercial Officer of Ultragenyx. “We are now focused on ensuring that all patients in the U.S. with LC-FAOD who might benefit from Dojolvi will have access to it.” To support access, Ultragenyx’s UltraCare® program helps patients and caregivers understand insurance coverage and assists them in finding financial support for Ultragenyx medicines, including Dojolvi, and for the administration of them. Dedicated in-house UltraCare Guides are available Monday through Friday from 9 a.m. to 8 p.m. Eastern Time at 888-756-8657. About 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 U.S. 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. LC-FAOD affect an estimated 2,000 to 3,500 children and adults in the United States. About Dojolvi Dojolvi is a highly purified, ...