FDA approves first drug in the US for rare genetic disease in children

The US Food and Drug Administration (FDA) has approved a drug from Fortress Biotech and Indian manufacturer Zydus Lifesciences for the treatment of a rare genetic disease in children, Fortress announced.

The medication, with the trade name Zycubo, is approved for the treatment of Menkes disease—a rare hereditary disorder in which patients are born with an inability to absorb copper from food. The condition is caused by mutations in a gene responsible for copper transport in the body.

The approval makes Zycubo the first drug on the US market intended for the treatment of this disease.

Symptoms of Menkes disease include sparse and discolored hair, seizures, and developmental delays. Without treatment, many patients do not survive beyond two to three years.

Under the terms of the agreement, Fortress Biotech is entitled to receive tiered royalties from net sales of Zycubo, as well as up to $129 million in combined payments upon achieving development and sales milestones for the drug.