U.S. FDA has assigned a target action date of January 28, 2022
Application based on results from the Phase 3 EXPLORER-HCM trial
PRINCETON, NJ, USA I March 19, 2021 IBristol Myers Squibb (NYSE: BMY) today announced that the U.S. Food and Drug Administration (FDA) has accepted its New Drug Application (NDA) for mavacamten, an investigational, novel, oral, allosteric modulator of cardiac myosin, for patients with symptomatic obstructive hypertrophic cardiomyopathy (oHCM). The FDA has assigned a Prescription Drug User Fee Act (PDUFA) goal date of January 28, 2022.
“HCM, which is the most common inherited heart disease, can be a chronic, debilitating, and progressive condition where patients may experience symptoms of shortness of breath, dizziness and fatigue as well as serious, life-altering complications, including heart failure, arrhythmias, stroke and sudden cardiac death,” said Roland Chen, M.D., Senior Vice President, Cardiovascular Development, Bristol Myers Squibb. “Today’s acceptance from the FDA puts us one step closer to having a highly targeted therapeutic approach for oHCM, as mavacamten is a first-in-class myosin inhibitor developed to address the underlying molecular defect of the disease. We are committed to supporting patients in need of HCM treatment and look forward to working with the FDA.”
The NDA submission was based on the results of the pivotal Phase 3 EXPLORER-HCM trial, which evaluated mavacamten in patients with symptomatic oHCM versus placebo. Results from the trial showed that mavacamten demonstrated a robust treatment effect, with clinically meaningful improvements in symptoms, functional status, and quality of life, as well as the ability to relieve left ventricular outflow tract obstruction. In the EXPLORER-HCM study all primary and secondary endpoints were met with statistical significance.
Mavacamten is an investigational therapy that is not approved for use in any country.
About Mavacamten
Mavacamten is a first-in-class, oral, allosteric modulator of cardiac myosin, under investigation for the treatment of conditions in which excessive cardiac contractility and impaired diastolic filling of the heart are the underlying cause. Mavacamten is thought to work by reducing cardiac muscle contractility by inhibiting excessive myosin-actin cross-bridge formation that results in hypercontractility, left ventricular hypertrophy and reduced compliance. In clinical and preclinical studies, mavacamten has consistently reduced biomarkers of cardiac wall stress, lessened excessive cardiac contractility, and increased diastolic compliance.
About EXPLORER-HCM
The EXPLORER-HCM Phase 3 trial enrolled a total of 251 patients with symptomatic (NYHA Class II or III), obstructive hypertrophic cardiomyopathy. All participants had measurable left ventricular outflow tract (LVOT) gradient (resting and/or provoked) ≥50 mmHg at baseline.
The primary endpoint for EXPLORER-HCM was a composite functional analysis designed to capture mavacamten’s effect on both symptoms and function. Secondary endpoints were changes from baseline to week 30 in postexercise LVOT gradient, pVO2, proportion of patients with at least one NYHA class improvement, and measures of patientreported outcomes. Additional endpoints included changes from baseline to Week 30 in echocardiographic indices, circulating biomarkers, cardiac rhythm patterns and accelerometry.
About Hypertrophic Cardiomyopathy
Hypertrophic cardiomyopathy, or HCM, is a chronic, progressive disease in which excessive contraction of the heart muscle and reduced ability of the left ventricle to fill can lead to the development of debilitating symptoms and cardiac dysfunction. HCM is estimated to affect one in every 500 people.
The most frequent cause of HCM is mutations in the heart muscle proteins of the sarcomere. In either obstructive or non-obstructive HCM patients, exertion can result in fatigue or shortness of breath, interfering with a patient’s ability to participate in activities of daily living. HCM has also been associated with increased risks of atrial fibrillation, stroke, heart failure and sudden cardiac death, with mortality among HCM patients shown to be approximately three-fold higher than the U.S. general population at similar ages.
There are currently approximately 160,000 to 200,000 people diagnosed with symptomatic obstructive HCM across the U.S. and EU, with no existing effective drug treatment options beyond limited symptomatic relief.
About Bristol Myers Squibb
Bristol Myers Squibb is a global biopharmaceutical company whose mission is to discover, develop and deliver innovative medicines that help patients prevail over serious diseases. For more information about Bristol Myers Squibb, visit us at BMS.com or follow us on LinkedIn, Twitter, YouTube, Facebook and Instagram.
SOURCE: Bristol Myers Squibb
Post Views: 41
U.S. FDA has assigned a target action date of January 28, 2022
Application based on results from the Phase 3 EXPLORER-HCM trial
PRINCETON, NJ, USA I March 19, 2021 IBristol Myers Squibb (NYSE: BMY) today announced that the U.S. Food and Drug Administration (FDA) has accepted its New Drug Application (NDA) for mavacamten, an investigational, novel, oral, allosteric modulator of cardiac myosin, for patients with symptomatic obstructive hypertrophic cardiomyopathy (oHCM). The FDA has assigned a Prescription Drug User Fee Act (PDUFA) goal date of January 28, 2022.
“HCM, which is the most common inherited heart disease, can be a chronic, debilitating, and progressive condition where patients may experience symptoms of shortness of breath, dizziness and fatigue as well as serious, life-altering complications, including heart failure, arrhythmias, stroke and sudden cardiac death,” said Roland Chen, M.D., Senior Vice President, Cardiovascular Development, Bristol Myers Squibb. “Today’s acceptance from the FDA puts us one step closer to having a highly targeted therapeutic approach for oHCM, as mavacamten is a first-in-class myosin inhibitor developed to address the underlying molecular defect of the disease. We are committed to supporting patients in need of HCM treatment and look forward to working with the FDA.”
The NDA submission was based on the results of the pivotal Phase 3 EXPLORER-HCM trial, which evaluated mavacamten in patients with symptomatic oHCM versus placebo. Results from the trial showed that mavacamten demonstrated a robust treatment effect, with clinically meaningful improvements in symptoms, functional status, and quality of life, as well as the ability to relieve left ventricular outflow tract obstruction. In the EXPLORER-HCM study all primary and secondary endpoints were met with statistical significance.
Mavacamten is an investigational therapy that is not approved for use in any country.
About Mavacamten
Mavacamten is a first-in-class, oral, allosteric modulator of cardiac myosin, under investigation for the treatment of conditions in which excessive cardiac contractility and impaired diastolic filling of the heart are the underlying cause. Mavacamten is thought to work by reducing cardiac muscle contractility by inhibiting excessive myosin-actin cross-bridge formation that results in hypercontractility, left ventricular hypertrophy and reduced compliance. In clinical and preclinical studies, mavacamten has consistently reduced biomarkers of cardiac wall stress, lessened excessive cardiac contractility, and increased diastolic compliance.
About EXPLORER-HCM
The EXPLORER-HCM Phase 3 trial enrolled a total of 251 patients with symptomatic (NYHA Class II or III), obstructive hypertrophic cardiomyopathy. All participants had measurable left ventricular outflow tract (LVOT) gradient (resting and/or provoked) ≥50 mmHg at baseline.
The primary endpoint for EXPLORER-HCM was a composite functional analysis designed to capture mavacamten’s effect on both symptoms and function. Secondary endpoints were changes from baseline to week 30 in postexercise LVOT gradient, pVO2, proportion of patients with at least one NYHA class improvement, and measures of patientreported outcomes. Additional endpoints included changes from baseline to Week 30 in echocardiographic indices, circulating biomarkers, cardiac rhythm patterns and accelerometry.
About Hypertrophic Cardiomyopathy
Hypertrophic cardiomyopathy, or HCM, is a chronic, progressive disease in which excessive contraction of the heart muscle and reduced ability of the left ventricle to fill can lead to the development of debilitating symptoms and cardiac dysfunction. HCM is estimated to affect one in every 500 people.
The most frequent cause of HCM is mutations in the heart muscle proteins of the sarcomere. In either obstructive or non-obstructive HCM patients, exertion can result in fatigue or shortness of breath, interfering with a patient’s ability to participate in activities of daily living. HCM has also been associated with increased risks of atrial fibrillation, stroke, heart failure and sudden cardiac death, with mortality among HCM patients shown to be approximately three-fold higher than the U.S. general population at similar ages.
There are currently approximately 160,000 to 200,000 people diagnosed with symptomatic obstructive HCM across the U.S. and EU, with no existing effective drug treatment options beyond limited symptomatic relief.
About Bristol Myers Squibb
Bristol Myers Squibb is a global biopharmaceutical company whose mission is to discover, develop and deliver innovative medicines that help patients prevail over serious diseases. For more information about Bristol Myers Squibb, visit us at BMS.com or follow us on LinkedIn, Twitter, YouTube, Facebook and Instagram.
SOURCE: Bristol Myers Squibb
Post Views: 41
