Alnylam’s rare disease drug shines in trial, paving way for a brand-new class of medicines
The positive results in the clinical trial help validate Alnylam's $7 billion market valuation and pave the way for more drugs based on RNAi technology.
The age of RNA interference drugs has arrived.
Alnylam Pharmaceuticals announced positive results Wednesday from a closely watched phase 3 clinical trial of patisiran, the company’s lead drug designed to treat a rare nerve disorder known as familial amyloid polyneuropathy (FAP.)
Most importantly, the safety profile of patisiran in the FAP study looks clean, assuaging concerns raised by toxicity reported
