Givosiran reduces annual attacks, ALA levels in acute hepatic porphyria

Alnylam Pharmaceuticals announced that givosiran, an investigational RNAi therapeutic in development for the treatment of acute hepatic porphyria, met its ENVISION phase 3 primary efficacy endpoint of reduction in the annualized rate of composite porphyria attacks compared with placebo, according to a press release and conference call.
“Patients living with [acute hepatic porphyria (AHP)] experience debilitating and sometimes life-threatening neurovisceral attacks as well as chronic disease manifestations which negatively impact their quality of life,” Akshay Vaishnaw, MD, PhD,

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