Alnylam on Wednesday unveiled positive data from a late-stage clinical trial for a drug candidate known as givosiran, the company’s second rare disease treatment to rely on the Nobel Prize-winning gene-silencing technique known as RNA interference.
Givosiran takes aim at a family of rare diseases known as acute hepatic porphyrias, which can cause abdominal issues that can be fatal. The study, which included 94 people in 18 countries, found that people on the drug had fewer attacks related to the genetic disorder over the course of six months. Five other secondary measurements also showed significant improvements.
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