The FDA has approved Oxlumo (lumasiran) as the first treatment for primary hyperoxaluria type 1 (PH1).
This rare genetic condition is caused by excess production of oxalate which is a substance consumed in food and produced in the body. People with PH1 produce far too much oxalate, which can combine with calcium to cause kidney stones, and this can lead to kidney damage and failure, and eventually damage the heart and a patient’s bones and eyes.
PH1 is the most common and severe form, and it affects an estimated one to three individuals per million in North America and Europe.
The drug Oxlumo works to reduce oxalate production. It was approved based on a clinical study: in the Oxlumo group, patients had a 65% reduction of oxalate in the urine, compared to an average 12% reduction in the placebo group. By the sixth month of the study, 52% of patients treated with Oxlumo reached a normal 24-hour urinary oxalate level while no patients treated with the placebo did.
Norman Stockbridge, Director of the Division of Cardiology and Nephrology in the FDA’s Center for Drug Evaluation and Research, said: “The approval of Oxlumo represents a great triumph of community involvement to address a rare disease. It is a result of input from patients, treating physicians, experts and sponsors at a patient-focused drug development meeting and through other collaborative efforts.”
This FDA approval follows the drug also being approved in Europe last week.
Conor Kavanagh
This is a syndicated feed from Pharmafile
