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# Alnylam Pharma NDA for Oxlumo Approved
- URL: https://www.fdaweb.com/alnylam-pharma-nda-for-oxlumo-approved/
- Published: 2020-11-24T12:00:00.000Z
- Updated: 2026-09-14T16:37:47.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5148156

FDA has approved an Alnylam Pharmaceuticals NDA for Oxlumo (lumasiran) as the first treatment for primary hyperoxaluria Type 1 (PH1), a rare genetic disorder. Primary hyperoxalurias (PHs) are caused by excess production of oxalate, a substance consumed in food and also produced by the body, an [FDA release](https://www.fda.gov/news-events/press-announcements/fda-approves-first-drug-treat-rare-metabolic-disorder?ref=fdaweb.com) says. “PH1 affects an estimated one to three individuals per million in North America and Europe and accounts for approximately 80% of PH cases,” it says.

Oxlumo works to decrease oxalate production. Approval was based on data from two studies in patients with PH1\. In the first study, patients in Oxlumo group had a 65% reduction of oxalate in the urine, compared to an average 12% reduction in the placebo group, the agency says. In the second study, oxalate patients had a 71% decrease in urinary oxalate by the sixth month of the study. The most common side effects of Oxlumo include injection site reaction and abdominal pain.