FDA Grants Orphan Drug Designation for Primary Mitochondrial Myopathies Treatment

  • Neuromuscular disease
  • peroxisome proliferator activated receptor
  • primary mitochondrial myopathies
  • REN001

The Food and Drug Administration (FDA) office of orphan products development has granted orphan drug designation to a peroxisome proliferator-activated receptor (PPAR) delta agonist, (REN001; Reneo Pharmaceuticals, San Diego, CA), for the treatment of primary mitochondrial myopathies (PMM). 

This selective PPAR delta agonist is in clinical development as a treatment for genetic myopathies, including PMM, which are a group of life-threatening diseases caused by genetic mutations impairing mitochondrial function. Individuals with PMM often have reduced muscle function adversely affecting daily functions and are faced with decreased life expectancy. There are no FDA-approved drugs for the treatment of PMM.

The 12-week clinical study (NCT03862846) was completed in participants with PMM and mitochondrial gene defects and a history of myopathy. Participants received the PPAR delta agonist orally, once-a-day, and a majority elected to enter an additional 36-week open-label extension study. The study looked at outcomes from walk tests and several symptom questionnaires. Safety data from the study suggests that the drug candidate was safe and well tolerated in participants with PMM. Preliminary walk-test data from this study is aiding in the design of a 200-participant clinical trial, which is expected to start in early 2021.

"Patients with primary mitochondrial myopathies have many aspects of their lives impacted," said Niall O'Donnell, PhD, chief officer executive of Reneo Pharmaceuticals. "The preliminary safety data and exploratory endpoint results from our first PMM clinical study has driven us to move forward, into a large, global clinical trial in PMM. We hope this will result in a great stride forward for patients with PMM."

On June 2nd, the FDA office of orphan products development granted orphan drug designation to PPAR delta agonist for the treatment of PMM. Last year, the FDA granted an orphan drug designation for PPAR delta agonist as treatment of fatty acid oxidation disorders. The PPAR delta agonist is also in clinical development for glycogen storage disease type V, also known as McArdle disease.

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