The US Food and Drug Administration (FDA) on Friday approved Stealth Biotherapeutics’ once-daily injection Forzinity for Barth syndrome, a rare genetic disorder that primarily affects males. It marks the first treatment ever cleared for the condition.

“This is the first treatment to be greenlit for the condition,” the FDA said in its announcement, according to Reuters. The regulator granted accelerated approval for patients weighing at least 30 kilograms.

Barth syndrome is a serious mitochondrial disease that often begins with severe heart failure in infancy and can cause premature death. Survivors face fatigue, poor stamina and exercise intolerance, the FDA noted.

The subcutaneous drug works by binding to the inner part of the mitochondria to improve its structure and function. Approval was based on improved strength of the leg muscle used to straighten the knee — a change the FDA deemed “reasonably likely to predict patient benefit, such as an ability to stand more easily or walk farther.”

Stealth will run additional studies to confirm the benefits. Under the FDA’s accelerated approval pathway, the agency can withdraw the drug if follow-up trials fail to show effectiveness.

An estimated 230 to 250 males worldwide were identified with Barth syndrome as of 2020, according to the National Institutes of Health.

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With inputs from Reuters