Debranching enzyme

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

Retrieved on: 
Tuesday, July 27, 2021
Hepatology, Enzymes, Inborn errors of carbohydrate metabolism, Medical specialties, Branches of biology, Glycogen storage disease type III, Glycogen storage disease, Glycogen debranching enzyme, Orphan drug, Debranching enzyme, Glycogen, AGL

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.

Key Points: 
  • 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.
  • 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.
  • The company is led by a management team experienced in the development and commercialization of rare disease therapeutics.