Alnylam Pharmaceuticals that it’s achieved full patient enrollment in its ILLUMINATE-C phase 3 study of lumasiran, an RNAi therapeutic targeting hydroxyacid oxidase 1 (HAO1) – the gene encoding glycolate oxidase (GO) – for the treatment of adults and youngsters with advanced primary hyperoxaluria type 1 (PH1). Enrollment was completed with 21 PH1 patients across 13 sites in 10 countries.
“We are pleased to possess completed enrollment within the ILLUMINATE-C phase 3 study,” said Pritesh J. Gandhi, PharmD., vice chairman and head , Lumasiran Program at Alnylam. “This is a crucial milestone to assist evaluate the efficacy and safety of lumasiran in patients with advanced PH1 including those experiencing systemic oxalosis and undergoing hemodialysis. We believe lumasiran has the potential to deal with the complete spectrum of PH1 disease severity and appearance forward to reporting topline results from this important study in mid-2021.”
Lumasiran was recently approved by the FDA for the treatment of PH1 to lower urinary oxalate levels in pediatric and adult patients. it had been also recently approved by the EMA for the treatment of PH1 altogether age groups. Lumasiran is marketed within the US and EU as Oxlumo.
ILLUMINATE-C may be a single arm, open-label, multinational phase 3 study to guage the security and efficacy of lumasiran in PH1 patients of all ages and advanced renal disease (eGFR = 45 mL/min/1.73m2 or elevated serum creatinine for patients
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