Ultragenyx Pharmaceutical, a biopharmaceutical company focused on the development and commercialization of novel products for rare and ultra-rare diseases, announced that Dojolvi (triheptanoin) is now commercially available. The US 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.
“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 US with LC-FAOD who might benefit from Dojolvi will have access to it.”
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 is included in newborn screening panels across the US 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 affects an estimated 2,000 to 3,500 children and adults in the United States.
Dojolvi is a highly purified, pharmaceutical-grade, odd-carbon medium-chain triglyceride consisting of three 7-carbon fatty acids on a glycerol backbone created via a multi-step chemical process. It is designed to provide medium-chain, odd-carbon fatty acids as an energy source and metabolite replacement for people with LC-FAOD.