Positive results from Phase III ILLUMINATE-C study of lumasiran in patients with advanced Primary Hyperoxaluria Type 1 (PH1) and severe renal impairment

Lumasiran is a RNAi therapeutic which inhibits production of oxalate, which directly contributes to the pathophysiology of PH1. In the study, lumasiran resulted in a substantial reduction in plasma oxalate from baseline in both dialysis-independent and -dependent patients.

SPS commentary:

Lumasiran is already approved in the EU and US for the treatment of PH1. The ILLUMINATE-C study however focused on patients with advanced renal disease (eGFR ≤ 45 mL/min/1.73m2 or elevated serum creatinine for patients <12 months of age). Based on these results, the Company plans to submit a Supplemental New Drug Application with the US FDA and a Type II Variation with the European Medicines Agency in late 2021.

The MHRA issued a positive Early Access to Medicines Scheme (EAMS) scientific opinion for lumasiran for the treatment of PH1 in all ages in July 2020, based on results of the ILLUMINATE A placebo-controlled RCT (n=39) and the ILLUMINATE B single-arm study (n=18).

Biospace Inc.

Resource links:

MHRA EAMS information