FDA approval of addition of trial data to label of cardiac myosin inhibitor

The U.S. Food and Drug Administration (FDA) approved the addition of positive data from the phase 3 VALOR-HCM trial to the U.S. Prescribing Information for mavacamten in patients with obstructive HCM.

Addition of positive data from the phase 3 VALOR-HCM trial to the U.S. Prescribing Information for mavacamten (2.5 mg, 5 mg 10 mg, 15 mg capsules) was approved by the FDA. These added data showed that treatment with mavacamten reduced the composite endpoint of eligibility for septal reduction therapy (SRT) at week 16 or the decision to proceed with SRT prior to or at week 16 in patients with obstructive HCM.

Last year, the FDA approved use of mavacamten for the treatment of adults with symptomatic NYHA class II-III obstructive HCM to improve functional capacity and symptoms, based on results from the phase 3 EXPLORER-HCM trial.

In the VALOR-HCM trial , patients with symptomatic obstructive HCM (NYHA class III-IV or class II with exertional syncope or near syncope) who met the 2011 ACC/AHA Guideline criteria and were referred for SRT were randomized to mavacamten (n=56) or placebo (n=56) for 16 weeks. The primary composite endpoint of patients eligible for SRT or the decision to proceed with SRT after 16 weeks of treatment was reduced in the mavacamten group compared with the placebo group; 82% of patients were no longer eligible for the surgical procedure and deciding not to proceed with SRT after 16 weeks of mavacamten.

No new adverse reactions were identified in VALOR-HCM.

Source: press release Bristol Myers Squibb, June 15, 2023