Orphan Drug Status Granted to Therapy for Primary Mitochondrial Myopathies


Orphan Drug Designation given to potential primary mitochondiral myopathies treatment.

A selective PPAR delta agonist, REN001 (Reneo Pharmaceuticals) has been granted an Orphan Drug Designation by the FDA for treating primary mitochondrial myopathies (PMM).

According to Reneo, REN001 is in clinical development as a treatment for genetic myopathies. Currently, there is no drug approved by the FDA for the treatment of PMM.

A recent 12- week study completed by Reneo involving the drug yielded positive results, according to the company. Over the course of the 12-week study, patients received REN001 orally once daily. The majority of patients, all of which were living with PMM, voluntarily entered an additional 36-week extension study. According to Reneo, REN001 was well tolerated and safe.

Results of the study will be used to design a 200-patient clinical trial which is expected to start in 2021.

“Patients with primary mitochondrial myopathies have many aspects of their lives impacted… 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,” CEO of Reneo Pharmaceuticals Niall O’Donnell, Ph.D. said in the press release.

Orphan Drug Designation is granted by the FDA office of Orphan Products development for novel drugs or biologics that treat a rare disease or condition that affects less than 200,000 patients in the United States. According to the press release, this qualifies sponsors for certain tax credits and up to 7 years of market exclusivity upon approval among other perks.

This is not the first Orphan Drug Designation REN001 has received. According to the company, it was granted the status for the treatment of fatty acid oxidation disorders.


Reneo Pharmaceuticals Completes Clinical Study in Primary Mitochondrial Myopathies and Receives FDA Orphan Drug Designation for REN001 [News Release]. San Diego, CA; June 23, 2020: Reneo Pharmaceuticals. https://reneopharma.com/reneo-pharmaceuticals-completes-clinical-study-in-primary-mitochondrial-myopathies-and-receives-fda-orphan-drug-designation-for-ren001/ Accessed June 24, 2020

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