FDA motions Alnylam's lumasiran into a PhIII short track, setting up a shot at an OK in '20
Alnylam $ALNY may be going solo on the development of rare disease drug lumasiran after Sanofi took a pass on its option, but the FDA has come through with a major assist. The company announced today that regulators have agreed on a Phase III study design that would accelerate — should all go well — its journey to approval.
For the trial designed to test lumasiran in the ultra-rare primary hyperoxaluria type 1 (PH1), the newly reached alignment allows Alnylam to adopt a primary endpoint based on a biomarker: the reduction of urinary oxalate at six months. The FDA has also sanctioned a study size of around 25 patients with PH1.
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