The Food and Drug Administration (FDA) has accepted for Priority Review the New Drug Application (NDA) for lumasiran (Alnylam Pharmaceuticals) for the treatment of primary hyperoxaluria type 1 (PH1).

Lumasiran is an investigational ribonucleic acid interference (RNAi) therapeutic that inhibits oxalate production by targeting hydroxyacid oxidase, thereby depleting glycolate oxidase. The NDA is supported by data from the phase 3 ILLUMINATE-A trial, which evaluated the efficacy and safety of lumasiran in 30 patients aged ≥6 years with PH1. 

Results showed that lumasiran met the primary end point of percent change from baseline, relative to placebo, in 24-hour urinary oxalate excretion averaged across Months 3 to 6 (P <.0001); statistical significance was also achieved across 6 hierarchically-tested secondary end points (P ≤.001), including the proportion of lumasiran patients that had near-normalization or normalization of urinary oxalate levels, compared with placebo. With regard to safety, lumasiran was found to be well tolerated and was not associated with any serious or severe adverse events.

A Prescription Drug User Fee Act (PDUFA) target date of December 3, 2020 has been set for this application.

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“Award of Priority Review status adds to the previous grants of Breakthrough Therapy and Pediatric Rare Disease Designations,” said Pritesh J. Gandhi, PharmD, Vice President and General Manager, Lumasiran Program at Alnylam. “Together, we believe these underscore the potential of lumasiran to address the underlying pathophysiology of PH1 and the urgent need for an FDA-approved treatment for this progressive, devastating disease where liver transplantation is currently the only treatment that addresses the root cause of disease.”

The Company is also expecting results from two phase 3 trials, ILLUMINATE-B and ILLUMINATE-C, evaluating lumasiran for the treatment of PH1, in mid-2020 and 2021, respectively.  

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