Article · Health news

FDA approves Camzyos for children

Health news Published · sources checked

The US FDA approved Camzyos (mavacamten) for children weighing at least 30 kg with symptomatic obstructive hypertrophic cardiomyopathy, on 30 September 2026.

Key points

  • The US FDA approved Camzyos (mavacamten) for children weighing at least 30 kg with symptomatic obstructive hypertrophic cardiomyopathy on 30 September 2026.
  • The FDA says it is the first treatment to improve functional capacity and symptoms in children with this condition.
  • Camzyos carries a boxed warning for the risk of heart failure and is available only through a restricted programme.
  • This is a US decision; UK availability is decided separately.

What was announced

On 30 September 2026 the US Food and Drug Administration (FDA) approved Camzyos (mavacamten) for children weighing at least 30 kg with symptomatic obstructive hypertrophic cardiomyopathy.

The FDA describes this as the first treatment to improve functional capacity and symptoms in children with the condition.

It describes the condition as inherited and causing a thickened heart muscle, with symptoms that can include shortness of breath, fatigue and an abnormal heart rhythm that can be life-threatening.

What it means

In the paediatric study, patients were randomly assigned to Camzyos or placebo, and Camzyos reduced the Valsalva left ventricular outflow tract gradient compared with placebo.

The FDA says adult data showed a similar reduction, and that it expects the drug to help paediatric patients weighing at least 30 kg.

Camzyos carries a boxed warning for the risk of heart failure and is available only through a restricted programme called the Camzyos Risk Evaluation and Mitigation Strategy (REMS).

This is a US decision; availability in the UK is decided separately by the MHRA and NICE.

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