Alnylam sets up 3rd potential RNAi approval with a showcase of positive lumasiran data
A week after nabbing a priority review, Alnylam has released pivotal data for their latest late-stage experimental drug — setting the stage for the RNA-targeting biotech to potentially win their 3rd FDA approval in as many years.
In the 39-person trial, patients with an ultra-rare liver condition PH1 (primary hyperoxaluria type 1) were given either placebo or the Alnylam drug lumasiran. Patients with PH1 overproduce a natural metabolic end-product, urinary oxalate, potentially leading to kidney stones and in many cases kidney failure at a young age, along with damage across the body.
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