Oxlumo Approved to Treat Rare Metabolic Disorder

November 23, 2020 – The U.S. FDA has approved OxlumoTM (lumasiran), manufactured by Alnylam Pharmaceuticals, to treat primary hyperoxaluria type 1 (PH1), lowering urinary oxalate levels, in pediatric and adult patients. Oxlumo is the first drug to receive FDA approval to treat PH1.

A rare metabolic disorder, PH1 is caused by a genetic mutation. The mutation causes oxalate, a naturally occurring substance present in many foods and as a waste product in the body, to accumulate in the kidneys and urinary tract. PH1 can be diagnosed as early as infancy, with signs and symptoms including frequent kidney stones, blood in the urine, urinary tract infections, and higher than normal levels of oxalate in the urine. Untreated, the condition can lead to life-threatening end-stage renal disease. It is important to begin treatment as soon as possible to preserve the patient’s kidney function.

In one clinical study, Oxlumo reduced urinary oxalate levels by an average of 65% in patients who were at least six years old. By the sixth month of the study, 52% of patients who received Oxlumo reached a normal 24-hour urinary oxalate level. Patients who received a placebo had only an average 12% reduction in urinary oxalate and did not reach a normal 24-hour urinary oxalate level. In a second study, 16 individuals under the age of six all received treatment with Oxlumo and saw on average a 71% decrease in urinary oxalate by the sixth month.

Oxlumo is given as a subcutaneous injection and must be administered by a healthcare professional. Under the recommended dosing, patients who weigh less than 10kg (22 pounds) should receive a loading dose of 6mg/kg once monthly for three months, followed by maintenance dosing of 3mg/kg once a month. Patients weighing 10kg to less than 20kg (44 pounds) should receive the same loading dose for three months, but then switch to a maintenance dose of 6mg/kg once every three months. Patients who weigh 20kg or more should start with a loading dose of 3mg/kg once monthly for three doses, followed by maintenance dosing of 3mg/kg once every three months.

Oxlumo has launched at a wholesale acquisition cost (WAC) of $55,000 per vial.