Alnylam Pharmaceuticals Inc. started a rolling submission of a new drug application to the U.S. Food and Drug Administration for approval of its experimental therapy lumasiran for a rare kidney disorder.
The Cambridge, Mass.-based biotech is developing lumasiran to treat primary hyperoxaluria type 1, or PH1, a disorder in which a substance called oxalate builds up 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.
Alnylam Vice President Pritesh Gandhi said the company expects to get an approval for lumasiran from the FDA in late 2020.
The application will include results from a recently completed phase 3 trial called Illuminate-A in which lumasiran performed better than placebo in reducing 24-hour urinary oxalate excretion. The company was evaluating the drug in patients with PH1, ages six and above.
Alnylam is also evaluating lumasiran in a phase 3 trial called Illuminate-B in patients with PH1 who are less than six years old.
The FDA also granted the drug pediatric rare disease designation for the treatment of PH1. Lumasiran holds orphan-drug tags from the U.S. and the European regulatory agencies in addition to the FDA's breakthrough-therapy designation and the European Medicines Agency's priority medicines designation.
Alnylam plans to seek approval for the drug from the EMA in early 2020.