FDA Grants Orphan Drug Designation to Reneo for REN001

Reneo Pharmaceuticals announced the U.S. Food and Drug Administration (FDA) Office of Orphan Products Development has granted Orphan Drug Designation to the company's lead drug candidate, REN001, for the treatment of fatty acid oxidation disorders (FAOD).

"There is a pressing need for effective treatments for patients suffering from the profound defects associated with FAOD, and we believe REN001 represents a promising new therapy. Orphan Drug Designation offers important incentives which help ensure that we can pursue this novel treatment for a set of diseases as rare as FAOD," said Niall O'Donnell, Ph.D., CEO of Reneo Pharmaceuticals.

REN001 is a selective PPAR delta agonist currently in development as a treatment for FAOD and other genetic myopathies. 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 U.S. clinical centers.

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

Orphan 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. The designation qualifies the sponsor for various development incentives of the Orphan Drug Act including tax credits for clinical research costs to advance the evaluation and development of products that demonstrate promise for the treatment of rare diseases. Orphan Drug Designation 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).

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