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Gene therapy for Haemophilia B approved in Europe

March 7, 2023

HEMGENIX® (etranacogene dezaparvovec) has received a conditional marketing authorisation from the European Commission.

UniQure, a company specialising in the development of gene therapies, has announced that the gene therapy Hemgenix, developed by uniQure and licensed by CSL Behring, has been approved in Europe for the treatment of severe and moderately severe haemophilia B.

Haemophilia B is a bleeding disorder caused by the lack of coagulation factor IX (FIX). It is perhaps best known for its influences in the European royal houses, such as in the severe case of Aleksei Romanov, heir to Nikolai II of Russia. Indeed, apart from very mild cases haemophilia B is almost solely seen in men due to the FIX gene being located to the X-chromosome, with women generally carrying a compensating healthy copy on their second X-chromosome.

In addition to external bleeds from cuts and wounds the patients suffer from internal bleeds for example to joints, which is extremely painful and can cause lasting damage. Haemophilia B is treated with intravenous injections of functional FIX, with patients suffering from the severe form sometimes requiring injections up to several times a week.

Hemgenix uses modified adeno-associated virus of serotype 5 (AAV5) to deliver a functional gene copy of FIX into the patient cells. The cells then read the instructions for making FIX in the delivered DNA and secrete the functional coagulation factor into the blood. Hemgenix is a one-time treatment, which in the clinical trials has demonstrated significantly reduced bleeds and eliminated the need for routine Factor IX injections for almost all patients.

Fimea has started the assessment of the therapeutic and economic value of Hemgenix, which will contribute to determining whether it will be offered by the public healthcare system in Finland.

Lähteet: uniQure, FIMEA, Duodecim

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