European Medicines Agency backs gene therapy to treat rare bleeding disorder


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The European Union’s health regulator on Friday said it had recommended granting a conditional marketing authorization for a gene therapy by Australian drugmaker CSL Ltd and partner uniQure N.V. , to treat haemophilia B, a rare bleeding disorder which is caused by genetic anomalies.

About one in 40,000 people are affected by the inherited disorder, caused by a gene mutation that hampers the body’s ability to make clotting protein factor IX.

If approved, the treatment, branded Hemgenix, will be the first gene therapy in the European Union for the condition that is usually treated by regular injections of factor IX, the European Medicines Agency said.

CSL had acquired exclusive global rights to Hemgenix in May last year from uniQure N.V.

In November, the U.S. Food and Drug Administration approved Hemgenix for the treatment of rare genetic blood clotting disorder, followed by CSL Ltd setting the list price of the therapy at $3.5 million making it the world’s most expensive treatment.


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