BMS Obtains FDA Approval for New HCM Treatment Camzyos
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United States Food and drug administration lit green Bristol Myers Squibb‘s Camzyos (mavacamten), the first FDA-approved cardiac myosin inhibitor that specifically targets the source of hypertrophic obstructive cardiomyopathy (HCM).
Obstructive HCM is a progressive disease in which the walls of the heart thicken, making it harder for the heart to expand normally and fill with blood. The mechanistic features of obstructive HCM are excessive formation of myosin actin cross-bridges and dysregulation of the super-relaxed state.
Camzyos is a selective allosteric and reversible inhibitor of cardiac myosin. The drug was designed to modulate the number of myosin heads that can enter the “on-actin” states of the nucleotide, which reduces the “likelihood of force-generating (systolic) and residual (diastolic) cross-bridge formation.” “, said the company. . The drug “shifts the overall myosin population to an energy-saving, recruitable, and super-relaxed state.”
The approval was data-driven from the phase III Explorer-HCM trial which showed that Camzyos could decrease the contractility of the heart by inhibiting the excessive formation of myosin-actin cross-bridges. After 30 weeks of treatment, patients in the phase III trial who received Camzyos showed greater cardiac improvement than those on placebo.
The primary endpoint of Explorer-HCM was defined as the proportion of patients who achieved either venous mixed oxygen tension improvement or NYHA class improvement. The data showed that after 30 weeks, 37% of Camzyos patients achieved the primary endpoint, compared to 17% of placebo patients.
Anjali T. Owens, medical director of the Center for Inherited Cardiac Disease and assistant professor of medicine at the Perelman School of Medicine in University of Pennsylvaniasaid Camzyos’ approval is a “significant milestone” for patients with appropriately symptomatic obstructive HCM.
Owens, principal investigator of the Explorer-HCM study, also noted that these patients and their families have been waiting for many years for a new treatment option for the progressive disease.
Samit Hirawat, executive vice president and medical director of global drug development at Bristol Myers Squibb, said Camzyos’ approval builds on “decades of cardiovascular leadership” and reflects the company’s commitment to treating people with cardiovascular disease.
“We are proud to bring this drug, the first of its kind, to patients, which may help address an unmet need in the United States in the symptomatic treatment landscape of obstructive HCM Class II-III from the NYHA,” Hirawat said in a statement.
Adverse effects occurred in more than 5% of patients in the Explorer-HCM trial, more frequently in the Camzyos group.
Camzyos comes with a boxed warning for the risk of heart failure. BMS noted that Camzyos reduces left ventricular ejection fraction (LVEF) and may cause heart failure due to systolic dysfunction. It is not recommended to treat patients with Camzyos if they have an LVEF below 55%. Camzyos will only be available on a restricted program under a Risk Assessment and Mitigation Strategy (REMS).