Lumasiran

Unassigned

New Medicines

Primary hyperoxaluria

Information

New molecular entity
Alnylam Pharmaceuticals
Alnylam Pharmaceuticals

Development and Regulatory status

Phase III Clinical Trials
Phase III Clinical Trials
Phase III Clinical Trials
Yes
Yes

Category

Metabolic disorders (09.08)
Silences the gene for glycolate oxidase to starve the pathway of oxalate production and prevent its associated pathology.[2,3]
One estimate places the prevalence of PH type I at 1-3 cases per 1,000,000 people in the general population and the incidence at 1 case per 120,000 live births per year in Europe.[1]
Primary hyperoxaluria
type 1 (PH1)
Subcutaneous injection