FDA Accepts Supplemental Application for Camzyos

The FDA has assigned a PDUFA goal date of June 16, 2023, for an expanded indication to reduce the need for septal reduction therapy (SRT), which is a procedure to treat hypertrophic cardiomyopathy.

The FDA has accepted Bristol Myers Squibb’s supplemental new drug application (sNDA) for Camzyos (mavacamten) for an expanded indication to reduce the need for septal reduction therapy (SRT), which is a procedure to treat hypertrophic cardiomyopathy. HCM is a chronic, progressive disease that make it hard for the heart to pump blood. 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,” Roland Chen, M.D., senior vice president and head of cardiovascular development, global drug development at Bristol Myers Squibb, said in a press release.

Related: FDA Approves Mavacamten for Hypertrophic Cardiomyopathy

The FDA approved Camzyos in April 2022. It addresses the excessive contraction of the heart that leads to severe disease where the blood flow is obstructed. It is thought to work by decreasing the number of actin-myosin cross-bridges, which reduces the heart muscle’s ability to contract.

The supplemental application is based on the VALOR-HCM phase 3 trial, which met its primary and all secondary endpoints with a high degree of statistical significance, with no new safety signals observed. The trial enrolled 112 enrolled patients who met guideline criteria for septal reduction therapy. The study included 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 was 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 at Week 16 in the mavacamten group compared with the placebo group.

Camzyos is only available through a restricted REMS program because of the risk of heart failure due to systolic dysfunction.