PITTSBURGH, July 2, 2020 /PRNewswire/ -- PANTHERx® Rare Pharmacy announces that it has been selected by Ultragenyx Pharmaceutical as a limited distribution partner for new drug DOJOLVI™ (triheptanoin). DOJOLVI™ (a highly purified, pharmaceutical-grade, synthetic seven carbon fatty acid triglyceride) is a specially designed synthetic triglyceride compound, which was developed to treat patients with long-chain fatty acid oxidation disorders (LC-FAOD). DOJOLVI™ provides these patients with a medium-length, odd-chain fatty acid that LC-FAOD patients can metabolize and serve as an alternative source of energy (by increasing intermediate substrates in the Krebs cycle, a key energy-generating process).
"PANTHERx Rare is proud to be chosen by Ultragenyx Pharmaceutical to deliver hope, white glove services, and DOJOLVI™ to people living with LC-FAOD," said Dr. Gordon Vanscoy, Chairman & CEO of PANTHERx. "Our unique RxARECARE™ Team structure ensures that new patients, and all of our patients, receive the level of superior quality, award-wining care that has become synonymous with PANTHERx. We look forward to making a difference in the lives of people living with LC-FAOD."
LC-FAODs 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 liver, muscle, and heart disease, which can lead to hospitalizations or early death. Currently, LC-FAOD patients are treated with the avoidance of fasting, low-fat/high carbohydrate diets, carnitine, and medium-chain triglyceride (MCT) oil, a medical food product. Despite current therapy, many patients have significant metabolic events including hospitalizations and mortality due to LC-FAOD.
About PANTHERx® Rare Pharmacy PANTHERx Rare Pharmacy, the largest independent and fastest growing specialty pharmacy in the United States, transforms lives by delivering medicine breakthroughs, clinical excellence, and access solutions to patients afflicted with rare and devastating conditions. Although the incidence is as common as diabetes, less than 7% of the 7,000 known rare and devastating disorders have an approved therapy. Changes in federal policy and advances in science have led to a surge in FDA orphan drug approvals, providing tremendous hope to the rare disease community.
PANTHERx was recently awarded Specialty Pharmacy of the Year by the National Association of Specialty Pharmacy (NASP) and earned the inaugural Accredited Distinction in Rare Diseases and Orphan Drugs from the Accreditation Commission for Health Care (ACHC). PANTHERx is a three-time winner of the prestigious MMIT Patient Choice Award including the 2020 honor. PANTHERx is headquartered in Pittsburgh, Pennsylvania, licensed in all 50 states, and holds accreditations from URAC, NABP, ACHC, and CPPA.