Orsini Pharmaceutical Services (“Orsini”), a leading independent national specialty pharmacy focused on patients with rare and complicated diseases, announced today that it has been selected by Ultragenyx Pharmaceutical Inc. as a limited distribution specialty pharmacy partner for Dojolvi™ (triheptanoin). Dojolvi™ is indicated 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).
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.
Orsini Chief Executive Officer Mike Fieri commented, “We are proud to expand our partnership with Ultragenyx and provide Dojolvi for people with LC-FAOD. High touch care coordination and collaboration drive our unique specialty pharmacy care model, and our LC-FAOD care team is focused on getting patients on therapy quickly and providing personalized and compassionate care.”
Source: Biospace
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