FDA expands Camzyos label to treat pediatric obstructive hypertrophic cardiomyopathy
The U.S. Food and Drug Administration approved an expanded indication for Bristol Myers Squibb’s cardiac myosin inhibitor, mavacamten (Camzyos), to include pediatric patients with symptomatic obstructive hypertrophic cardiomyopathy who weigh at least 30 kg. The decision, announced on September 30 2026, is based on the phase‑3 SCOUT‑HCM trial that enrolled 44 adolescents aged 12 to < 18 years. The trial showed a mean reduction in Valsalva LVOT gradient of about 48 mm Hg versus placebo and no patient experienced a left‑ventricular ejection fraction below 50 %.
Why it matters
The approval gives Bristol Myers Squibb a new, FDA‑cleared market for Camzyos in children, potentially increasing sales and providing physicians with the only approved therapy for pediatric oHCM. The company’s press release notes that the drug has already been prescribed to more than 25,000 patients in the United States, suggesting a sizable existing prescriber base that can now be extended to younger patients.
Key facts
- 1The FDA announced the expanded indication on September 30 2026. hcplive.com
- 2The label now covers pediatric patients weighing at least 30 kg (66 lb). pharmacytimes.com
- 3SCOUT‑HCM enrolled 44 adolescents (23 receiving mavacamten, 21 receiving placebo). hcplive.com
- 4At week 28, the least‑squares mean difference in Valsalva LVOT gradient was –48.0 mm Hg (mavacamten vs placebo). hcplive.com
- 5No patient in the trial had a left‑ventricular ejection fraction drop below 50 %. hcplive.com
- 6The drug remains available only through the Camzyos REMS program with required echocardiographic monitoring. pharmacytimes.com
Open questions
- Material 2 states that pediatric safety was evaluated in patients weighing at least 35 kg, while the label permits use at 30 kg.
- Material 2 reports a Valsalva LVOT gradient change of –48.5 mm Hg for mavacamten versus –0.5 mm Hg for placebo, a slight numeric difference from the –48.0 mm Hg difference cited in material 1.
Summary written by AlphAI from 4 of 4 sources. Not investment advice. Figures are as stated by the linked sources.