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US FDA approves Regeneron’s ultra-rare blood disease drug


(Reuters) -Regeneron Pharmaceuticals said on Friday the U.S. health regulator approved its drug to treat a rare blood disease.

The company said its drug, Veopoz, was approved in adult and pediatric patients 1 year of age and older with the life-threatening CHAPLE disease.

People with CHAPLE disease have mutated CD55 gene, which regulates the body’s mechanism for destroying microbes. Without proper gene regulation, the mechanism may start attacking normal cells of the body.

In most patients, this results in a range of potentially life-threatening symptoms beginning in early childhood.

The company did not immediately respond to a Reuters request for comment on the drug’s price.

(Reporting by Vaibhav Sadhamta, Khushi Mandowara and Pratik Jain in Bengaluru; Editing by Shilpi Majumdar)

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