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Alnylam Shows Efficacy, Safety With Lumasiran In Vulnerable Pediatric Patients

ILLUMINATE-B Is The Second Successful Phase III Study In Primary Hyperoxaluria 1

 
• By Joseph Haas

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.

Cute Little Girl Sits on a Hospital Bed and Talks with Friendly Woman Doctor Taking Blood Test. Children's Hospital Pediatric Ward. Top Quality Health Care.
Alnylam hopes lumasiran will greatly improve the treatment paradigm in PH1

Alnylam Pharmaceuticals Inc.’s lumasiran already is on track to become the first approved drug therapy for the ultra-rare renal disorder primary hyperoxaluria 1 (PH1), but top-line data the biotech unveiled on 30 September from a second Phase III study were the first ever safety and efficacy evaluation of an RNA-interference therapy in patients six years and younger.

Lumasiran is under US Food and Drug Administration review with an action date of 3 December, on the strength of data from the Phase III ILLUMINATE-A trial

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