Investigational Treatment for Fatty Acid Oxidation Disorders Gets Orphan Drug Designation

The Food and Drug Administration (FDA) has granted Orphan Drug designation to REN001 (Reneo Pharmaceuticals) for the treatment of fatty acid oxidation disorders.

REN001 is a selective peroxisome proliferator-activated receptor (PPAR) delta agonist currently being investigated in a phase 1b trial involving 12 patients with confirmed fatty acid oxidation disorders. The primary outcome measure of the study is to assess the safety and tolerability of REN001; results are expected in summer 2020.

“There is a pressing need for effective treatments for patients suffering from the profound defects associated with [fatty acid oxidation disorders], and we believe REN001 represents a promising new therapy,” said Niall O’Donnell, PhD, CEO of Reneo Pharmaceuticals.

Orphan drug designation is granted to treatments for rare conditions that affect fewer than 200,000 people in the US. Fatty acid oxidation disorders are a group of potentially life-threatening genetic disorders that affect the ability of mitochondria to utilize long chain fatty acids as an energy source. Muscle injury and breakdown, cardiomyopathy and reduced exercise capacity are some of the symptoms associated with these disorders.

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Last month Ultragenyx submitted a New Drug Application to the FDA for triheptanoin, a potential treatment for long-chain fatty acid oxidation disorders. 

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