First gene_therapy_haemophilia_B EMA has recommended granting a conditional marketing authorisation in the European Union (EU) for Hemgenix (etranacogene dezaparvovec), the the first gene therapy for the treatment of severe and moderately severe haemophilia B in adults who do not have factor IX inhibitors (auto-antibodies produced by the immune system which make factor IX medicines less effective). 

Haemophilia B is an inherited disorder characterised by an increased bleeding tendency due to a partial or complete deficiency of coagulation factor IX, a protein needed to produce blood clots to stop bleeding. Medicines currently authorised to treat haemophilia B aim to prevent bleeding episodes or to treat bleeding episodes that may occur during surgery or in emergencies even when patients are on regular therapy. Patients require a lifelong routine treatment regimen, representing an unmet medical need for new therapeutic approaches.  

This represents a great advance for a better quality of life for these patients, however, there is still no treatment option for the paediatric population affected by this disorder, which still represents a significant unmet medical need.  

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By Published On: December 23rd, 2022Categories: News0 Comments