Hemophilia B Gene Therapy Among Five Drugs On Track For EU-Wide Approval

 
• By Ian Schofield

Alongside CSL/Behring/uniQure’s Hemgenix (etranacogene dezaparvovec), the European Medicines Agency has given the green light to new drugs for liver cancer, NSCLC, Pompe disease and multiple sclerosis. But it turned down omburtamab for treating the rare cancer, neuroblastoma.  

Blood Sample for Factor IX. Blood coagulation testing.
Hemophilia B is a genetic disorder caused by a missing or defective factor IX clotting protein • Source: Shutterstock

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