FDA approves Bristol Myers Squibb's Camzyos for pediatric oHCM

The U.S. Food and Drug Administration has approved an expanded indication for Bristol Myers Squibb's (NYSE: BMY) Camzyos (mavacamten) to include pediatric patients with symptomatic obstructive hypertrophic cardiomyopathy (oHCM), the company announced. The therapy is now approved for adults and pediatric patients weighing 30 kg (66 lbs) or more.

The approval is based on results from the Phase 3 SCOUT-HCM trial, a randomized, double-blind, placebo-controlled study that enrolled 44 adolescent patients aged 12 to under 18 years. The trial compared Camzyos (n=23) against placebo (n=21) over 28 weeks.

Camzyos met the trial's primary endpoint, showing a statistically significant reduction in Valsalva left ventricular outflow tract (LVOT) gradient at Week 28 compared with placebo. The least-squares mean difference from placebo was -48.0 mmHg (95% CI: -67.7 to -28.3; P<0.0001).

In the trial, no patients experienced left ventricular ejection fraction (LVEF) below 50%, and no adverse events led to treatment discontinuation. Serious adverse events occurred in 2 patients in each group — 9% in the Camzyos group and 10% in the placebo group. No new adverse reactions were identified beyond the safety profile observed in adults.

Camzyos was first approved by the FDA in 2022 for adults with symptomatic NYHA Class II-III oHCM. According to the company, the drug has been prescribed by more than 5,000 healthcare providers to over 25,000 patients in the U.S. Bristol Myers Squibb said the pediatric data is being reviewed by multiple global regulatory authorities for potential pediatric indications.

Camzyos carries a boxed warning for the risk of heart failure due to systolic dysfunction and is available only through a restricted Risk Evaluation and Mitigation Strategy (REMS) program. The drug is contraindicated with strong CYP2C19 inhibitors and moderate to strong CYP2C19 or CYP3A4 inducers.

The above information is based on a press release issued by Bristol Myers Squibb.

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