mavodelpar

Mavodelpar is a selective peroxisome proliferator-activated receptor delta (PPARδ) agonist and a small molecule drug under investigation for the treatment of rare mitochondrial diseases, particularly primary mitochondrial myopathies (PMM) and long-chain fatty acid oxidation disorders (LCFAOD), including long-chain 3-hydroxyacyl-CoA dehydrogenase (LCHAD) deficiency. It acts by activating PPARδ, which plays a key role in regulating genes involved in energy metabolism and mitochondrial function. Mavodelpar has demonstrated the ability to improve cellular bioenergetics and alleviate mitochondrial dysfunction in preclinical models and patient-derived cells. The drug has received Fast Track designation from the FDA for LCHAD deficiency. It is being developed by Reneo Pharmaceuticals[1][2][3][4][5][6][7].

Other names

mavodelpar sodium