Reducing Statin-induced Myopathy
Novel treatment for HMGCR hereditary and statin-induced myopathy
Limb Girdle Muscular Dystrophies (LGMD) encompass a group of rare autosomal diseases characterized by proximal muscle weakness. Despite their shared attribute, LGMDs have diverse genetic origins and varied disease progression, ranging from mild disability to complete loss of mobility. Symptoms can involve skeletal muscle atrophy, contractures, weakness in different muscle groups, and complications such as cardiomyopathy and respiratory difficulties. With over 50 genetic loci and 30 known associated genes, LGMDs exhibit significant genetic heterogeneity. Among these, a novel form of LGMD has been identified which mimics the myopathy induced by statins, commonly prescribed for dyslipidemia (high cholesterol).
Currently, supportive care is the primary approach for managing LGMDs, but ongoing genetic therapy trials offer hope for future treatments.
Additionally, statins may lead to statin-associated muscle symptoms (SAMS) affecting up to 30% of users, with severe cases resulting in rhabdomyolysis or immune-mediated necrotizing myositis (IMNM) and potentially causing disability or death.
Inventors
Prof. Ohad Birk, Soroka Medical Center
Dr. Yuval Yogev, Soroka Medical Center
Contact info
Sari Prutchi Sagiv PhD. Director of Pharma and Diagnostics
For further information please contact:
sari@mor-research.comThe inventors have developed a novel method for treating myopathy, specifically targeting myopathies associated with inhibition or disruptions of the mevalonate pathway.
They synthesized and purified a molecule named D-mevalonolactone and have shown that oral administration of this compound is effective in treating human HMGCR muscular dystrophy and statin-induced myopathy in mice, without any significant adverse effects. In vivo experiments, including a murine model, have demonstrated the protective effects of mevalonolactone against myotoxicity induced by high-dose statin treatment.
In addition, a clinical study has been performed in a patient suffering from HMGCR-LGMD, under compassion treatment procedure. The patient suffered from severe limb girdle myopathy and total dependence on respiratory support. During treatment, there was improvement in muscle strength noticed among all muscle groups, with up to 200% improvement in muscle strength.
LGMD is an orphan indication and development of a drug can benefit of orphan status – subsidies, tax credits, and waivers and profitability.
The statin market has huge potential. The global statin market size reached US$ 14.3 Billion in 2021 and expected to reach US$ 17.5 Billion by 2027. Atorvastatin, rosuvastatin, lovastatin and simvastatin are the most common types of statins available in the market. As the most prescribed drug class in the world, with a high rate of adverse events, this indication may be useful for many patients.