The US Food and Drug Administration ( FDA ) has approve the most expensive drug in the world : Hemgenix , a gene therapy intervention for the rare transmissible disease hemophilia B.

Hemophilia B is a rare hemorrhage upset , triggered by the deficiency of a blood protein calledFactor IX . Treatments are available for the disorderliness , include even endovenous infusion to help with clotting and keep bleeding episode . These treatments themselves are incredibly expensive over a life .

" Total grownup lifetime costs per patient with stark and moderately severe [ hemophilia B ] were $ 21,086,607 for [ received half - life Factor IX ] prophylaxis , $ 22,987,483 for [ extended half - life story Factor IX ] prophylaxis , and $ 20,971,826 for on - requirement [ Factor IX ] intervention , " one study issue in theJournal of Medical Economicsfound , call it a " significant economic encumbrance " .

This young therapy , created by pharmaceutic company CSL Behring , is agene therapythat can be given as a single VD to cross your lifetime .

" Hemgenix comprise of a viral transmitter gestate a gene for clotting Factor IX , " the FDA explicate in astatement . " The gene is expressed in the liver to make Factor IX protein , to increase bloodline levels of Factor IX and thereby limit bleeding episode . "

Studies line up that the therapy cut the hemorrhage rate in patient by 54 percentage liken to their baseline annual bleed rate .

“ Gene therapy for hemophilia has been on the horizon for more than two 10 . Despite advancement in the treatment of hemophilia , the bar and discourse of leech episodes can adversely impact mortal ’ tone of biography , ” Peter Marks , music director of the FDA ’s Center for Biologics Evaluation and Research , say in the assertion about Tuesday ’s approval .

“ Today ’s approval furnish a new treatment option for patients with Hemophilia B and represents important progress in the development of innovative therapies for those experiencing a high burden of disease associate with this form of hemophilia . ”

Though the drug is cheaper than the price of other treatment over a patient role ’s lifetime , it still costsa hefty $ 3.5 million , making it the most expensive drug in the world . The cost is a concern , though some analysts think the nature of the consideration may make patients take the discussion anyway .

“ While the price is a little gamey than expect , I do think it has a probability of being successful because 1 ) exist drugs are also very expensive and 2 ) hemophilia patients constantly live in fear of bleeds , ” biotechnology investor and CEO of Loncar Investments Brad Loncar toldBloomberg .

“ A gene therapy product will be invoke to some . ”