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 the U.S. Food and Drug Administration (FDA) has approved Dojolvi™ (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). LC-FAOD are 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 is a highly purified, synthetic, 7-carbon fatty acid triglyceride specifically designed to provide medium-chain, odd-carbon fatty acids as an energy source and metabolite replacement for people with LC-FAOD.
“With today’s FDA approval, patients living with this serious, unpredictable, and often catastrophic disease now have an approved therapy. Many patients with long-chain fatty acid oxidation disorders have difficult lives with frequent hospitalizations and major medical events despite the best current care. Now these patients have an approved treatment as an option to help manage their disease,” said Camille L. Bedrosian, M.D., Chief Medical Officer of Ultragenyx. “We are grateful to the patients, caregivers, families, dietitians and doctors involved for helping to make this community milestone possible, and we are committed to ensuring that all patients in the U.S. 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 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.
“FDA approval of Dojolvi for the treatment of long-chain fatty acid oxidation disorders is a huge milestone for patients and their families, as it is the first such medication,” said Dr. Jerry Vockley, Professor of Human Genetics and Chief of Medical Genetics at Children's Hospital of Pittsburgh of UPMC. “While we have been able to identify these disorders at birth for many years, treatment options have been limited. Dojolvi brings hope as a new tool now available to clinicians taking care of these patients. Rare disease research, especially development of new therapies, is an arduous and time consuming process, and today’s announcement represents the culmination of nearly 20 years of work on the part of numerous researchers, clinicians, and patients. I’m tremendously excited to have been a part of this process.”
Ultragenyx expects Dojolvi to be available to patients in the next 30 days. 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.
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.
Ultragenyx licensed exclusive worldwide rights to triheptanoin from Baylor Research Institute in 2013.
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).
IMPORTANT SAFETY INFORMATION
WARNINGS AND PRECAUTIONS
Feeding Tube Dysfunction
Intestinal Malabsorption in Patients with Pancreatic Insufficiency
Pancreatic Lipase Inhibitors
USE IN SPECIFIC POPULATIONS
Pregnancy and Lactation
PATIENT COUNSELING INFORMATION
The patient or caregiver should:
Please see full Prescribing Information, including the Patient Information leaflet, for a complete discussion of the risks associated with Dojolvi.
Ultragenyx is a biopharmaceutical company committed to bringing to patients novel products for the treatment of serious rare and ultra-rare genetic diseases. The company has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease.
The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx’s strategy is predicated upon time- and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency.
For more information on Ultragenyx, please visit the company’s website at www.ultragenyx.com.
Source: Ultragenyx Pharmaceutical Inc.