FDA Grants Fast Track Designation for Reneo Pharma's Mavodelpar (REN001) for LCHAD Deficiency Treatment

FDA Grants Fast Track Designation for Reneo Pharmaceuticals' Mavodelpar (REN001) for LCHAD Deficiency Treatment

FDA Grants Fast Track Designation for Reneo Pharma's Mavodelpar (REN001) for LCHAD Deficiency Treatment

Reneo Pharmaceuticals, Inc., a clinical-stage pharmaceutical company focused on developing and commercialising therapies for patients with rare genetic mitochondrial diseases granted Fast Track designation from the U.S. Food and Drug Administration (FDA) for mavodelpar (REN001) to treat long-chain 3-hydroxyacyl-CoA dehydrogenase (LCHAD) deficiency, one of the most common genotypes in long-chain fatty acid oxidation disorder (LC-FAOD) patients.

Publish Date: 31-01-2023 Source: Reneo Pharmaceuticals, Inc.

Fatty Acid Oxidation Disorders (FAODs) are a group of inherited metabolic conditions that result in an accumulation of fatty acids and a decrease in cell energy metabolism. Each fatty acid oxidation disorder is caused by a specific enzyme defect in the fatty acid metabolic pathway and has an impact on dietary and stored fat utilisation. The presentation of a FAOD varies depending on the disorder, but common elements may be observed and ultimately require similar treatment. Initial presentations of the FAODs in the neonatal period with severe symptoms include cardiomyopathy, while liver dysfunction and hypoketotic hypoglycemia are common during infancy and childhood. Fatty Acid Oxidation Disorders (FAODs) are all inherited in an autosomal recessive manner. Patients will have inherited two copies of a FAOD with a mutation or variant in the DNA sequence. Each pregnancy will have a one-in-four chance of a child being affected, a one-in-two chance of being an unaffected carrier, and a one-in-four chance of being unaffected and not a carrier.

According to the study, there is an estimated collective incidence of 0.85 to 1.12 cases in every 5,000 to 10,000 births, although the individual prevalence varies significantly.
MCAD is the most common of the fatty acid oxidation disorders, affecting one in every 10,000 to 20,000 births. LCHAD and VLCAD are rare disorders that affect one in every 100,000 births. There appears to be a mild form of SCAD deficiency that is quite common, but the clinical significance of this condition is unknown.

However, the current Fatty Acid Oxidation Disorders (FAODs) treatment market share, market uptake, and attribute analysis concerning the most potential emerging therapies (UX007, REN001, etc..) has been provided under the market outlook section of the study covering 8 MM countries; The United States, EU5 (Germany, Spain, France, Italy, UK) Japan and China.

In terms of pharmacologic therapies, several pharmaceutical products are being approved and under different phases of development for the Fatty Acid Oxidation Disorders (FAODs) treatment. The key companies in the advanced development stage are Ultragenyx Pharmaceutical Inc, Reneo Pharma Ltd, etc., targeting FAODs.

Based on solid domain and business knowledge, Thelansis Knowledge Partners has published the market outlook forecast report on Fatty Acid Oxidation Disorders (FAODs) to provide a clear understanding of disease area background, epidemiology, current and future competitions, the country-specific standard of care, and the complete market forecast for 2022 to 2032.

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