Camzyos (mavacamten), a first-in-class heart disease drug, was approved by the FDA on April 28, 2022 to treat symptomatic obstructive hypertrophic cardiomyopathy (HCM), a genetic condition that causes an enlarged dysfunctional heart and is the leading cause of sudden cardiac death among young people. Marketed by Bristol Myers Squibb (BMS), this drug was discovered by the biotech company MyoKardia, which was co-founded by BioFrontiers CSO and MCDB Distinguished Professor Leslie Leinwand. MyoKardia was later acquired by BMS in a multi-billion dollar deal. Technology pioneered in Leslie’s lab was key to the discovery and directly led to initial identification of this molecule. HCM impacts 1 in 500 Americans, but until now there has been no primary treatment, nor has there ever been a drug approved that binds and modulates the protein myosin, which is the motor driving heart contraction and which can cause HCM when mutated. It is estimated that there are millions of patients worldwide who could benefit from this therapy.
Read reaction from the HCM patient community: https://4hcm.org/the-mavacamten-marathon/