FDA Grants Orphan Drug Designation to Treatment for Fatty Acid Oxidation Disorders


Officials from the FDA’s Office of Orphan Products Development have granted Reneo Pharmaceuticals’ REN001 Orphan Drug Designation for the treatment of fatty acid oxidation disorders (FAOD).1

REN001 is a selective PPAR delta agonist that is currently in development as a treatment for FAOD. The ongoing clinical trial is a 12-week, Phase 1b open-label study designed to evaluate safety and tolerability of REN001 in adult patients with FAOD in several United States clinical centers. REN001 is intended to improve cellular energy metabolism by enhancing mitochondrial function and potentially increasing the number of mitochondria.1,2

FAOD are a group of rare, potentially life-threatening genetic disorders that affect the ability of mitochondria to utilize long chain fatty acids as an energy source. Muscle injury, muscle breakdown, cardiomyopathy, and reduced exercise capacity are symptoms of FAOD, which greatly impact patient quality of life and can result in frequent hospitalizations. There are currently no approved drugs for treatment of patients with FAOD.1

Orphan Drug Designation is granted by The FDA Office of Orphan Products Development to novel drugs or biologics that treat a rare disease or condition affecting fewer than 200,000 patients in the U.S. It also conveys up to seven years of market exclusivity upon approval of the drug, as well as the ability to apply for annual federal grants and the waiver of filing fees through the Prescription Drug User Fee Act (PDUFA).1


  • FDA grants orphan drug designation to Reneo Pharmaceuticals for REN001 for treatment of fatty acid oxidation disorders [news release]. San Diego, CA: September 20, 2019; Reneo Pharmaceuticals. https://prnmedia.prnewswire.com/news-releases/fda-grants-orphan-drug-designation-to-reneo-pharmaceuticals-for-ren001-for-treatment-of-fatty-acid-oxidation-disorders-300922249.html. Accessed September 23, 2019.
  • REN001. Reneo Pharmaceuticals website. https://reneopharma.com/. Published 2019. Accessed September 23, 2019.

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