FDA approves mavacamten for hypertrophic cardiomyopathy
Bristol Myers Squibb has announced that the U.S. Food and Drug Administration (FDA) has approved the supplemental New Drug Application (sNDA) to include positive data from the Phase 3 VALOR-HCM study in the U.S. Prescribing Information for CAMZYOS® (mavacamten, 2.5 mg, 5 mg, 10 mg, 15 mg capsules). The additional data demonstrates that treatment with CAMZYOS significantly reduces the composite endpoint of guideline-based eligibility for septal reduction therapy (SRT) at Week 16 or the decision to proceed with SRT prior to or at Week 16.
CAMZYOS was previously approved by the FDA in the previous year based on the results from the Phase 3 EXPLORER-HCM trial. It was approved for the treatment of symptomatic New York Heart Association (NYHA) class II-III obstructive hypertrophic cardiomyopathy (HCM) in adults to improve functional capacity and symptoms.
Catherine Owen, Senior Vice President and General Manager, U.S. Commercial, Bristol Myers Squibb, stated, "CAMZYOS is the first and only FDA-approved cardiac myosin inhibitor that specifically targets the underlying source of the disease and is redefining the treatment landscape for symptomatic NYHA class II-III obstructive HCM. Results from the Phase 3 VALOR-HCM study reinforce the data from the Phase 3 EXPLORER-HCM trial and further strengthen the clinical profile of CAMZYOS. We are proud to offer this important therapy to patients."
However, it is important to note that the U.S. Prescribing Information for CAMZYOS carries a Boxed Warning regarding the risk of heart failure. CAMZYOS reduces left ventricular ejection fraction (LVEF) and can cause heart failure due to systolic dysfunction. Echocardiogram assessments of LVEF are required before and during treatment with CAMZYOS. Initiation of CAMZYOS is not recommended in patients with LVEF less than 55%. The use of CAMZYOS is contraindicated with certain cytochrome P450 inhibitors or inducers due to the increased risk of heart failure.
Hypertrophic cardiomyopathy is the most common inherited heart disease, affecting an estimated one in 200 to one in 500 people in the U.S. Obstructive HCM, the most common type of HCM, is caused by dysfunction in the sarcomere, leading to a thickened heart muscle that obstructs or reduces blood flow from the heart to the rest of the body. Patients with severe symptoms and a dynamic left ventricular outflow tract (LVOT) gradient may be eligible for septal reduction therapy (SRT) to reduce the thickness of the septal wall and alleviate obstruction.
Anjali T. Owens, Medical Director of the Center for Inherited Cardiac Disease and Associate Professor of Medicine at the Perelman School of Medicine at the University of Pennsylvania, and VALOR-HCM trial investigator and executive committee member, highlighted the need for more treatment options for patients recommended for SRT. She said, "SRT is an invasive surgical or catheter-based procedure and is typically available at comprehensive HCM treatment centers. In order to provide broader access to treatment for those patients whose obstructive HCM becomes so advanced that guidelines recommend SRT, more treatment options are needed."
The Phase 3 VALOR-HCM study enrolled patients with symptomatic obstructive HCM who met the criteria for SRT. The results showed that CAMZYOS significantly reduced the primary composite endpoint, with 82% of patients no longer eligible for SRT and deciding not to proceed with the surgical procedure after 16.
Source:
U.S. food and Drug Administration approves addition of positive data from Phase 3 VALOR-HCM study to CAMZYOS® (mavacamten) label. (n.d.). Bms.com. Retrieved June 16, 2023
