The top-line data in the ultra-rare disease are also the first to demonstrate safety and efficacy of an RNAi therapy in patients six years and younger.

Execs are hoping having positive results in two studies will help raise funds for continued development. But high placebo results have caused concern for investors.

The latest drug development news and highlights from our US FDA Performance Tracker.

Alnylam will take on the global development and commercialization of investigational rare disease therapy lumasiran as Sanofi declines to opt-in to its development, and the US biotech prepares to commercialize its first RNAi therapeutic, pencilled in for later this year.