As with its first two approved RNAi therapies, Alnylam will negotiate value-based agreements with payers, but for Oxlumo, approved in pediatric patients, it will offer a patient need adjustment.

Alnylam shows that lumasiran offers similar safety and efficacy in primary hyperoxaluria type 1 patients who are six or younger as it does in older patients. US and EU regulatory decisions loom.

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.

Building on data first reported last December, lumasiran appears on pace for approval by the December 2020 action date, but Dicerna still may show its drug is best-in-class.