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Reneo Receives Orphan Drug Designations from the EMA for REN001 for the Treatment of LCHAD Deficiency and MELAS Syndrome and Moves Forward with Clinical Trials

A global clinical trial is slated to begin for early next year to evaluate REN001 for the treatment of primary mitochondrial myopathies (PMM), including MELAS syndrome


For LCHAD deficiency and other fatty acid oxidation disorders (FAOD), a Phase 1b clinical trial is currently recruiting and expanding globally and an international observational study is planned to begin this fall


SAN DIEGO, September 9, 2020 – Reneo Pharmaceuticals announced today that 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.


For more info on current clinical trials for REN001, see


About EU Orphan Drug Designation

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.


About Reneo

Reneo Pharmaceuticals is a clinical stage pharmaceutical company focused on the development of therapies for patients with genetic mitochondrial diseases. Many of these diseases are associated with deficiencies in mitochondrial energy production. The company’s goal is to improve daily function and quality of life of patients suffering from these diseases, most specifically, by improving how their mitochondria work, preserving muscle function and preventing muscle injury, weakness and wasting. The experienced team of drug development experts, who have collaborated on many successful programs, is dedicated and passionate about finding effective therapies for these complex rare diseases.

Source: Reneo Pharmaceuticals, Inc.