US FDA approves Fortress Bio and Zydus’ treatment for a rare pediatric disease

Jan 13 (Reuters) – The U.S. Food and Drug Administration approved Fortress Biotech and Indian drugmaker Zydus Lifesciences’ drug for children with a rare genetic disorder that impairs copper absorption, Fortress said on Tuesday.

Shares of Fortress Bio rose more than 13% in premarket trading.

The drug, branded as Zycubo, is approved to treat Menkes disease, where patients are born without the ability to absorb copper from their diet. It is caused by mutations in a particular gene that is responsible for the transport of copper throughout the body.

The approval makes it the first drug on the market to treat this disease in the United States.

Symptoms of this condition include sparse and de-pigmented hair, seizures and developmental delays. If left untreated, many patients die between the ages of two and three years.

The disease primarily affects male infants. According to the companies, recent estimates suggest a prevalence of 1 in 34,810 to as high as 1 in 8,664 live male births.

Fortress is eligible to receive tiered royalties on net sales of Zycubo and up to $129 million in aggregate development and sales milestones from Zydus.

The drug, chemically known as CUTX-101, is an under-the-skin injectable formulation of copper histidinate, a copper replacement that can be injected directly into the body to avoid absorption through the gastrointestinal tract.

It helps increase copper levels in the blood, to improve body and brain function.

The approval was based on data showing Zycubo improved overall survival in patients who received early treatment, with a nearly 80% reduction in the risk of death compared with those who were untreated.

Current treatments include daily copper injections.

(Reporting by Padmanabhan Ananthan and Sneha S K in Bengaluru; Editing by Devika Syamnath)