Alnylam Pharmaceuticals Inc. will file for regulatory approval of lumasiran in early 2020 after the medicine bested placebo in a late-stage study of patients with a rare kidney disorder.
Lumasiran was evaluated against placebo in a phase 3 trial called Illuminate-A in patients ages six and above with primary hyperoxaluria type 1, or PH1.
PH1 is a rare disorder that causes a buildup of a substance called oxalate in the kidneys. Oxalate is normally filtered through the kidneys and excreted in the urine, but when combined with calcium, the nutrient forms the main component of kidney and bladder stones.
The therapy met the main goal of the study by being better than placebo in reducing 24-hour urinary oxalate excretion. The trial also achieved statistically significant results for all six secondary goals. The company said in a Dec. 17 press release that no serious or severe adverse events were observed in the trial and lumasiran was generally well-tolerated.
Alnylam plans to file for regulatory approvals for lumasiran in the U.S. and EU in early 2020. The drug previously received orphan-drug status in the U.S. and EU, breakthrough-therapy designation from the U.S. Food and Drug Administration, and a priority medicines designation from the European Medicines Agency.
Detailed results from the Illuminate-A trial will be presented at a conference in March 2020.
The company is also evaluating lumasiran in a phase 3 trial called Illuminate-B in patients with PH1 who are less than six years old. Results are expected in mid-2020. Alnylam will also issue results from another global phase 3 trial, known as Illuminate-C, in PH1 patients of all ages with advanced renal disease in 2021.