Reneo Pharmaceuticals Inc, a clinical-stage pharmaceutical company, received Fast Track designation from the U.S. Food and Drug Administration (FDA) for Mavodelpar (REN001) in the treatment of long-chain 3-hydroxyacyl-CoA dehydrogenase (LCHAD) deficiency, a genotype often reported 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 J. Flesher, president and chief executive officer of Reneo Pharmaceuticals.
Read more about LCFAOD treatments
“We appreciate the continued collaboration with the FDA and look forward to their partnership in advancing the LCFAOD program, which will include patients with LCHAD as well as other genotypes,” he added.
Mavodelpar is a potential therapeutic option for patients with rare mitochondrial disease. The company recently finished a natural history study called FORWARD and an open-label study assessing mavodelpar in LCFAOD. Mavodelpar is also being assessed in the clinical trial STRIDE in patients with primary mitochondrial myopathies who also have mitochondrial DNA defects. The STRIDE study results are anticipated to be available in the fourth quarter of this year.
LCFAOD are rare genetic metabolic disorders caused by nuclear DNA (nDNA) alterations, preventing the metabolic breakdown of long-chain fatty acids. The most severe cases of LCFAOD are reportedly diagnosed within the first few days or weeks of life and can lead to encephalopathy, liver dysfunction, lethargy, a high risk for sudden death in young patients, and hypoglycemia.
On the other hand, older patients usually experience muscle weakness, muscle aches, exercise intolerance, or rhabdomyolysis, damaging the heart and kidneys and causing permanent disability or even death.
Reference
Reneo pharmaceuticals receives fast track designation from the FDA for Mavodelpar (REN001) in a genotype of long-chain fatty acid oxidation disorder (LC-FAOD). Reneo Pharmaceuticals Inc; Published online January 31, 2023.
