U.S. FDA has assigned a target action date of June 16, 2023
Application based on results from the Phase 3 VALOR-HCM study
PRINCETON, NJ, USA I October 21, 2022 I Bristol Myers Squibb (NYSE: BMY) today announced that the U.S. Food and Drug Administration (FDA) has accepted its supplemental new drug application (sNDA) for CAMZYOS® (mavacamten) for an expanded indication to reduce the need for septal reduction therapy (SRT). CAMYZOS is currently FDA approved for the treatment of adults with symptomatic New York Heart Association (NYHA) class II-III obstructive hypertrophic cardiomyopathy (HCM) to improve functional capacity and symptoms. The FDA assigned a Prescription Drug User Fee Act (PDUFA) goal date of June 16, 2023.
“Currently, it is recommended that many patients with severe symptomatic obstructive hypertrophic cardiomyopathy undergo SRT. This often requires either an open-heart surgical procedure or septal ablation procedure – both specialized care options,” said Roland Chen, MD, senior vice president and head of cardiovascular development, Global Drug Development at Bristol Myers Squibb. “The approval of CAMZYOS earlier this year marked a significant milestone for patients. FDA acceptance of the filing for this expanded indication has the potential to strengthen the profile of CAMZYOS, while further reinforcing our commitment to delivering transformative cardiovascular therapies to patients.”
The sNDA submission was based on the results of the Phase 3 VALOR-HCM study, a randomized, double-blind, placebo-controlled study, which evaluated CAMZYOS in patients with symptomatic, obstructive HCM (NYHA class III-IV) who met the 2011 ACC/AHA guideline criteria for SRT and who have been referred for an invasive procedure. VALOR-HCM met its primary and all secondary endpoints with a high degree of statistical significance, with no new safety signals observed.
About the Phase 3 VALOR-HCM Trial
VALOR-HCM (NCT04349072) is a randomized, double-blind, placebo-controlled, multicenter Phase 3 study of patients with symptomatic, obstructive HCM (NYHA class III-IV) who meet guideline criteria for septal reduction therapy (SRT) and have been referred for an invasive procedure. The study enrolled 112 patients randomized on a 1:1 basis to receive mavacamten or placebo. VALOR-HCM includes three treatment periods over 128 weeks: a 16-week placebo-controlled period, a 16-week active treatment period where all patients received mavacamten and a 96-week long-term extension period where all patients received mavacamten.
The primary endpoint of VALOR-HCM is a composite of the number of patients who decide to proceed with SRT prior to or at Week 16 and the number of patients who remain SRT-guideline eligible (LVOT gradient of ≥50mmHg and NYHA Class III-IV) at Week 16 in the mavacamten group compared with the placebo group. Key secondary endpoints include impact on exercise gradient LVOT, NYHA Class and Kansas City Cardiomyopathy Questionnaire (KCCQ) and biomarkers at Week 16.
About Obstructive Hypertrophic Cardiomyopathy
Obstructive hypertrophic cardiomyopathy (obstructive HCM) is a chronic, progressive disease in which excessive contraction of the heart muscle and reduced ability of the left ventricle to fill can make it difficult for blood to circulate to the rest of the body, leading to the development of debilitating symptoms and cardiac dysfunction. HCM can be hereditary and can develop at any age. Patients are typically diagnosed in their 40s or 50s, and as many as 50% of patients have a hereditary predisposition.
In obstructive HCM, which is the most common type of HCM, the left ventricular outflow tract (LVOT) where blood leaves the heart becomes obstructed by the enlarged heart muscle. As a result, obstructive HCM has also been associated with increased risks of atrial fibrillation, stroke, heart failure and, although rare, sudden cardiac death. The most frequent cause of obstructive HCM is mutations in the heart muscle proteins of the sarcomere. Obstructive HCM is estimated to affect 400,000-600,000 people worldwide, however many patients remain undiagnosed and/or asymptomatic.
About CAMZYOS (mavacamten)
CAMZYOS (mavacamten) is the first and only cardiac myosin inhibitor approved by the U.S. Food and Drug Administration (FDA) indicated for the treatment of adults with symptomatic New York Heart Association (NYHA) class II-III obstructive hypertrophic cardiomyopathy (HCM) to improve functional capacity and symptoms. CAMZYOS is an allosteric and reversible inhibitor selective for cardiac myosin. CAMZYOS modulates the number of myosin heads that can enter “on actin” (power-generating) states, thus reducing the probability of force-producing (systolic) and residual (diastolic) cross-bridge formation. Excess myosin actin cross-bridge formation and dysregulation of the super-relaxed state are mechanistic hallmarks of HCM. CAMZYOS shifts the overall myosin population towards an energy-sparing, recruitable, super-relaxed state. In HCM patients, myosin inhibition with CAMZYOS reduces dynamic LVOT obstruction and improves cardiac filling pressures.
About CAMZYOS REMS Program
CAMZYOS is only available through a restricted program called the CAMZYOS REMS Program because of the risk of heart failure due to systolic dysfunction. Notable requirements of the CAMZYOS REMS Program include the following:
- Prescribers must be certified by enrolling in the REMS Program.
- Patients must enroll in the REMS Program and comply with ongoing monitoring requirements.
- Pharmacies must be certified by enrolling in the CAMZYOS REMS Program and must only dispense to patients who are authorized to receive CAMZYOS.
- Wholesalers and distributors must only distribute to certified pharmacies.
Further information is available by telephone at 1-833-628-7367.
INDICATION
CAMZYOS (mavacamten) is indicated for the treatment of adults with symptomatic New York Heart Association (NYHA) class II-III obstructive hypertrophic cardiomyopathy (HCM) to improve functional capacity and symptoms.
Please see US Full Prescribing Information, including Boxed WARNING and Medication Guide.
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: 26
U.S. FDA has assigned a target action date of June 16, 2023
Application based on results from the Phase 3 VALOR-HCM study
PRINCETON, NJ, USA I October 21, 2022 I Bristol Myers Squibb (NYSE: BMY) today announced that the U.S. Food and Drug Administration (FDA) has accepted its supplemental new drug application (sNDA) for CAMZYOS® (mavacamten) for an expanded indication to reduce the need for septal reduction therapy (SRT). CAMYZOS is currently FDA approved for the treatment of adults with symptomatic New York Heart Association (NYHA) class II-III obstructive hypertrophic cardiomyopathy (HCM) to improve functional capacity and symptoms. The FDA assigned a Prescription Drug User Fee Act (PDUFA) goal date of June 16, 2023.
“Currently, it is recommended that many patients with severe symptomatic obstructive hypertrophic cardiomyopathy undergo SRT. This often requires either an open-heart surgical procedure or septal ablation procedure – both specialized care options,” said Roland Chen, MD, senior vice president and head of cardiovascular development, Global Drug Development at Bristol Myers Squibb. “The approval of CAMZYOS earlier this year marked a significant milestone for patients. FDA acceptance of the filing for this expanded indication has the potential to strengthen the profile of CAMZYOS, while further reinforcing our commitment to delivering transformative cardiovascular therapies to patients.”
The sNDA submission was based on the results of the Phase 3 VALOR-HCM study, a randomized, double-blind, placebo-controlled study, which evaluated CAMZYOS in patients with symptomatic, obstructive HCM (NYHA class III-IV) who met the 2011 ACC/AHA guideline criteria for SRT and who have been referred for an invasive procedure. VALOR-HCM met its primary and all secondary endpoints with a high degree of statistical significance, with no new safety signals observed.
About the Phase 3 VALOR-HCM Trial
VALOR-HCM (NCT04349072) is a randomized, double-blind, placebo-controlled, multicenter Phase 3 study of patients with symptomatic, obstructive HCM (NYHA class III-IV) who meet guideline criteria for septal reduction therapy (SRT) and have been referred for an invasive procedure. The study enrolled 112 patients randomized on a 1:1 basis to receive mavacamten or placebo. VALOR-HCM includes three treatment periods over 128 weeks: a 16-week placebo-controlled period, a 16-week active treatment period where all patients received mavacamten and a 96-week long-term extension period where all patients received mavacamten.
The primary endpoint of VALOR-HCM is a composite of the number of patients who decide to proceed with SRT prior to or at Week 16 and the number of patients who remain SRT-guideline eligible (LVOT gradient of ≥50mmHg and NYHA Class III-IV) at Week 16 in the mavacamten group compared with the placebo group. Key secondary endpoints include impact on exercise gradient LVOT, NYHA Class and Kansas City Cardiomyopathy Questionnaire (KCCQ) and biomarkers at Week 16.
About Obstructive Hypertrophic Cardiomyopathy
Obstructive hypertrophic cardiomyopathy (obstructive HCM) is a chronic, progressive disease in which excessive contraction of the heart muscle and reduced ability of the left ventricle to fill can make it difficult for blood to circulate to the rest of the body, leading to the development of debilitating symptoms and cardiac dysfunction. HCM can be hereditary and can develop at any age. Patients are typically diagnosed in their 40s or 50s, and as many as 50% of patients have a hereditary predisposition.
In obstructive HCM, which is the most common type of HCM, the left ventricular outflow tract (LVOT) where blood leaves the heart becomes obstructed by the enlarged heart muscle. As a result, obstructive HCM has also been associated with increased risks of atrial fibrillation, stroke, heart failure and, although rare, sudden cardiac death. The most frequent cause of obstructive HCM is mutations in the heart muscle proteins of the sarcomere. Obstructive HCM is estimated to affect 400,000-600,000 people worldwide, however many patients remain undiagnosed and/or asymptomatic.
About CAMZYOS (mavacamten)
CAMZYOS (mavacamten) is the first and only cardiac myosin inhibitor approved by the U.S. Food and Drug Administration (FDA) indicated for the treatment of adults with symptomatic New York Heart Association (NYHA) class II-III obstructive hypertrophic cardiomyopathy (HCM) to improve functional capacity and symptoms. CAMZYOS is an allosteric and reversible inhibitor selective for cardiac myosin. CAMZYOS modulates the number of myosin heads that can enter “on actin” (power-generating) states, thus reducing the probability of force-producing (systolic) and residual (diastolic) cross-bridge formation. Excess myosin actin cross-bridge formation and dysregulation of the super-relaxed state are mechanistic hallmarks of HCM. CAMZYOS shifts the overall myosin population towards an energy-sparing, recruitable, super-relaxed state. In HCM patients, myosin inhibition with CAMZYOS reduces dynamic LVOT obstruction and improves cardiac filling pressures.
About CAMZYOS REMS Program
CAMZYOS is only available through a restricted program called the CAMZYOS REMS Program because of the risk of heart failure due to systolic dysfunction. Notable requirements of the CAMZYOS REMS Program include the following:
- Prescribers must be certified by enrolling in the REMS Program.
- Patients must enroll in the REMS Program and comply with ongoing monitoring requirements.
- Pharmacies must be certified by enrolling in the CAMZYOS REMS Program and must only dispense to patients who are authorized to receive CAMZYOS.
- Wholesalers and distributors must only distribute to certified pharmacies.
Further information is available by telephone at 1-833-628-7367.
INDICATION
CAMZYOS (mavacamten) is indicated for the treatment of adults with symptomatic New York Heart Association (NYHA) class II-III obstructive hypertrophic cardiomyopathy (HCM) to improve functional capacity and symptoms.
Please see US Full Prescribing Information, including Boxed WARNING and Medication Guide.
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: 26
