Even though there is currently no cure for hemophilia there are different ways to treat the condition. These include prophylaxis or preventative treatment, and recombinant factor concentrates, used depending on the type and severity of the condition.
1. Prophylaxis – regular preventive treatment, is typically administered three times a week.
2. Extended half-life (EHL) factor concentrates – alternative to prophylaxis and only available for hemophilia A and B these can also be used as a preventive measure. EHL factor concentrates work for longer periods after being administered, which is what researchers call ‘increased half-life’.
New long-lasting treatments are being developed and approved by regulatory authorities worldwide. Research pipeline is centered around the three main approaches:
- Fc fusion
- PEGylation (polyethylene glycol conjugation)
- Albumin fusion
Two new longer-lasting factor concentrates developed with an Fc fusion technology were just approved by the U.S. Food and Drug Administration (FDA). These are:
- Alprolix BIIB029 – for hemophilia B (approved in March 2014)
- Eloctate BIB031 – for hemophilia A (approved in June 2014)
Long-lasting factor concentrates being synthesized through PEGylation are:
- N9-GP – for hemophilia B (likely to receive a U.S. license next year)
- N8-G9 – for hemophilia A
- BAX-385 – for hemophilia A
- BAY 94-9027 – for hemophilia A
Another long-lasting product developed using the albumin fusion technique is:
- CSL654 – for hemophilia A
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