AstraZeneca, Merck Drug Approved for Painful Childhood Ailment

(Bloomberg) -- AstraZeneca Plc and Merck & Co. received U.S. approval for a drug to treat a painful, damaging condition that afflicts children with a rare nerve-cell cancer.

The drug, called Koselugo, is the first to be approved to treat children suffering from neurofibromatosis type 1 who have tumors within the protective sheaths surrounding their nerves, according to a statement.

The approval adds to AstraZeneca’s string of successes in finding uses for its many cancer drugs, such as Tagrisso, a top seller that recently produced positive results in patients whose lung cancer cells have a specific gene mutation. Cambridge, England-based AstraZeneca and U.S.-based Merck are also partners on Lynparza, which targets cancers with a different type of genetic abnormality.

AstraZeneca received a priority review voucher through a U.S. program intended to encourage development of new treatments for rare childhood diseases. Companies with vouchers can get quick regulatory review of experimental treatments, and some have sold for more than $100 million.

As many as half of patients with neurofibromatosis type 1 suffer from plexiform neurofibromas. These growths can cause pain, disfigurement and difficulties with movement, breathing and digestion, according to AstraZeneca’s statement.

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