No­var­tis' ip­ta­co­pan meets PhI­II test in fight against rare blood dis­ease

No­var­tis de­clared a Phase III suc­cess for its drug ip­ta­co­pan for the treat­ment of parox­ys­mal noc­tur­nal he­mo­glo­bin­uria, a rare dis­or­der in which flawed red blood cells are at­tacked by the im­mune sys­tem, forc­ing pa­tients to get blood trans­fu­sions.

The study, called AP­POINT-PNH, showed that pa­tients who got 200 mg of ip­ta­co­pan twice dai­ly achieved clin­i­cal­ly mean­ing­ful he­mo­glo­bin-lev­el in­creas­es of 2 g/dL or more from base­line with­out the need for blood trans­fu­sions at 24 weeks, the com­pa­ny an­nounced in a press re­lease Thurs­day.

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