The FDA granted Reneo Pharmaceuticals’ (NASDAQ:RPHM) mavodelpar (REN001) fast track designation 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).
“Receiving this fast track designation for a second indication continues to solidify mavodelpar as a potential therapeutic option for patients with rare mitochondrial disease,” Gregory Flesher, president and CEO of Reneo, said in a statement.
The company 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) in patients with primary mitochondrial myopathies with mitochondrial DNA defects. The company expects topline data from the STRIDE study in the fourth quarter of this year.
