Reneo Pharmaceuticals announced the European Medicines Agency's (EMA) Committee for Orphan Medicinal Products has granted the company orphan drug designation for REN001, an investigational medicine in development for the treatment of LCHAD deficiency, a fatty acid oxidation disorder (FAOD), and MELAS syndrome, a primary mitochondrial myopathy (PMM). REN001 was previously granted orphan drug designation by the U.S. Food and Drug Administration (FDA) for the treatment of both FAOD and PMM.

"Receiving orphan drug designation for REN001 in the European Union, based on our clinical trial results, is an important milestone that underscores the treatment need that exists for patients with genetic myopathies," said Niall O'Donnell, Ph.D., CEO of Reneo Pharmaceuticals. "We are grateful for the support of our clinical sites who are planning to take part in the upcoming FAOD and PMM clinical trials."

Reneo is rapidly advancing REN001, a selective PPAR delta agonist, in clinical development as a potential treatment for genetic myopathies, including FAOD and PMM. These myopathies are genetic disorders causing impaired energy production in muscle cells leading to reduced muscle function that adversely impacts daily functions and decreases life expectancy. There are no EMA or FDA approved drugs for treating PMM.

Reneo recently announced the completion of a 12-week clinical study in PMM patients with mitochondrial gene defects and a history of myopathy. Data from the study suggest that REN001 was safe and well tolerated in PMM patients. Preliminary positive functional data from this study is aiding in the design of an international clinical trial for PMM, which is expected to start in early 2021. For FAOD patients, a Phase 1b clinical trial is currently recruiting in the U.S. and is expanding to additional international sites, and an observational study is planned to begin later this year.

Orphan drug designation in the European Union (EU) is granted by the European Commission based on a positive opinion issued by the EMA Committee for Orphan Medicinal Products. To qualify, an investigational medicine must be intended to treat a seriously debilitating or life-threatening condition that affects fewer than five in 10,000 people in the EU, and there must be sufficient non-clinical or clinical data to suggest the investigational medicine may produce clinically relevant outcomes. EMA orphan drug designation provides companies with certain benefits and incentives, including clinical protocol assistance, differentiated evaluation procedures for Health Technology Assessments in certain countries, access to a centralized marketing authorization procedure valid in all EU member states, reduced regulatory fees and 10 years of market exclusivity.