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Emmaus withdraws EU market application for sickle cell treatment

Emmaus Life Sciences Inc. has pulled the marketing application for its sickle cell treatment Xyndari from the European Medicines Agency due to an opinion from the regulator that the medication was not effective.

The European regulator's Committee for Medicinal Products for Human Use, known as CHMP, said the Torrance, Calif.-based company's application did not show that Xyndari could reduce the number of sickle cell disease crises or hospital visits.

The treatment holds an orphan designation from the European Commission since 2012.

"Because we have demonstrated the efficacy of Xyndari, as supported by the data from the trials conducted, we are disappointed in the CHMP's position," Emmaus CEO Yutaka Niihara said in a Sept. 19 release. "We are seriously considering a decentralized approval procedure on a country-by-country basis."

In the U.S., the drug is named Endari and has been approved by the U.S. Food and Drug Administration for patients more than 5 years old.

Emmaus was delisted from the Nasdaq stock exchange just over a week prior to announcing the European application withdrawal.