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Ultragenyx Receives Orphan Drug Designation from FDA and European Commission for UX053, an Investigational mRNA-therapy for the Treatment of Glycogen Storage Disease Type III
Phase 1/2 study is expected to initiate enrollment in the second half of 2021 NOVATO, Calif., July 27, 2021 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc.
About this update from Ultragenyx Pharmaceutical Inc.
[{"type":"text","content":"Phase 1/2 study is expected to initiate enrollment in the second half of 2021\nNOVATO, Calif., July 27, 2021 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultra-rare genetic diseases, today announced that the U.S. Food and Drug Administration (FDA) and the European Commission have granted Orphan Drug Designation for UX053 for the treatment of Glycogen Storage Disease Type III (GSDIII), a metabolic disease that affects more than 10,000 patients in the developed world. Enrollment in a Phase 1/2 clinical trial evaluating the safety, tolerability, and efficacy of UX053 in adults with GSDIII is expected to begin in the second half of 2021. About Orphan Drug Designation The FDA Orphan Drug Designation program provides orphan status to drugs and biologics that are intended for the safe and effective treatment, diagnosis, or prevention of rare diseases that affect fewer than 200,000 people in the U.S. Among the benefits of orphan designation in the U.S. are seven years of market exclusivity following FDA approval, waiver or partial payment of application fees, and tax credits for clinical testing expenses conducted after orphan designation is received. The European Commission grants orphan drug designation for medicinal products intended to treat life-threatening or chronically debilitating conditions that affect fewer than five in 10,000 people in the European Union and when no satisfactory method of diagnosis, prevention or treatment of the condition can be authorized. The designation provides certain benefits and incentives in the EU, including protocol assistance, fee reductions, and ten years of market exclusivity once the medicine is on the market. About Glycogen Storage Disease Type III Glycogen Storage Disease Type III, or GSDIII, a disease caused by a glycogen debranching enzyme (AGL) deficiency that results in glycogen accumulation in the liver and muscle. GSDIII can cause hepatomegaly, hypoglycemia, hyperlipidemia, some progressive liver cirrhosis, and skeletal and cardiac muscle disease. There are no approved treatment options for GSDIII and the current standard of care is a strict diet, including frequent doses of cornstarch, to reduce the risk of hypoglycemia. GSDIII affects more than 10,...