FDA Grants Fast Track Designation to Reneo’s Mavodelpar in a Genotype of LC-FAOD

Rare Daily Staff

The U.S. Food and Drug Administration has granted Fast Track designation to Reneo Pharmaceuticals’ mavodelpar (for long-chain 3-hydroxyacyl-CoA dehydrogenase (LCHAD) deficiency, one of the predominant genotypes in patients with long-chain fatty acid oxidation disorder (LC-FAOD).

LC-FAOD are a group of rare, genetic metabolic disorders caused by mutations or deletions in the nuclear DNA. These genetic alterations prevent the body from breaking down long-chain fatty acids during metabolism. The most severe cases of LC-FAOD are diagnosed within the first few days or weeks of life. Young patients often present with a severe energy deficit that results in lethargy, liver dysfunction, hypoglycemia, encephalopathy, and high risk for sudden death. Older patients usually present with muscle weakness, exercise intolerance, muscle aches, or rhabdomyolysis which can damage the heart and kidneys and cause permanent disability or even death.

Mavodelpar is a potent and selective peroxisome proliferator-activated receptor delta (PPARδ) agonist currently in clinical development for two rare genetic mitochondrial diseases that typically present with myopathy and have high unmet medical needs: primary mitochondrial myopathies (PMM) and long-chain fatty acid oxidation disorders (LC-FAOD).

Reneo recently completed a natural history study (FORWARD) and an open label study evaluating mavodelpar in patients with LC-FAOD that included multiple genotypes, including the LCHAD genotype. Mavodelpar is also being evaluated in a pivotal clinical trial (STRIDE study) in patients with primary mitochondrial myopathies (PMM) with mitochondrial DNA (mtDNA) defects. The company expects topline data from the STRIDE study in the fourth quarter of this year.

The FDA’s Fast Track regulatory process is designed to facilitate and expedite the development of investigational treatments that demonstrate a potential to address unmet medical needs in serious or life-threatening conditions. Programs with FDA Fast Track designation can benefit from early and frequent communication with the FDA in addition to a rolling submission of the marketing application.

“Receiving this Fast Track designation for a second indication continues to solidify mavodelpar as a potential therapeutic option for patients with rare mitochondrial disease,” said Gregory Flesher, president and CEO of Reneo Pharmaceuticals. “We appreciate the continued collaboration with the FDA and look forward to their partnership in advancing the LC-FAOD program which will include patients with LCHAD as well as other genotypes.”

Photo: Gregory Flesher, president and CEO of Reneo Pharmaceuticals