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Bezastad is a brand name for bezafibrate, a synthetic small molecule belonging to the fibrate class. It functions as a pan-peroxisome proliferator-activated receptor (PPAR) agonist, targeting all three isoforms: PPARα, PPARβ/δ, and PPARγ. While traditionally approved and used as a lipid-lowering agent for the treatment of hyperlipidemia and dyslipidemia, it is being repurposed for mitochondrial diseases, including mitochondrial myopathies. Its therapeutic potential in these conditions stems from its ability to activate the PPAR/PGC-1α pathway, which induces mitochondrial biogenesis and improves mitochondrial respiratory function. Clinical research, such as Phase 2 trials conducted by the Univ.-Klinik für Kinder- und Jugendheilkunde in Austria, has investigated its efficacy in improving exercise tolerance and clinical rating scales in patients with mitochondrial dysfunction.
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