Alnylam Shows Efficacy, Safety With Lumasiran In Vulnerable Pediatric Patients
ILLUMINATE-B Is The Second Successful Phase III Study In Primary Hyperoxaluria 1
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.
You may also be interested in...
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.
The end of the year should see a flurry of new products reaching the market to break new commercial ground or shake up their respective therapeutic areas. Here, Scrip takes a look at 12 of the more interesting approvals expected in the coming months, with the help of analysts at Biomedtracker.
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.