Initial results of Phase 3 trial of lumasiran in kids are promising

Alnylam Pharmaceuticals, Inc. announced positive preliminary results from the company’s ILLUMINATE-A Phase 3 study of OXLUMO® (lumasiran), an RNAi therapeutic targeting hydroxy acid oxidase 1 – the gene encoding glycolate oxidase, for the treatment of primary hyperoxaluria type 1, a ultra-rare genetic disease characterized by overproduction of oxalate in the liver.1 The data was released at the virtual meeting of Pediatric Academic Societies.

The study was a 6-month, randomized, double-blind, placebo-controlled, global, multicentre Phase 3 study that evaluated the efficacy and safety of lumasiran in 39 patients aged 6 years and older with a documented diagnosis of primary hyperoxaluria type 1. Patients were randomized 2: 1 to receive either 3 monthly doses of the drug or placebo, followed by 3-monthly doses of 3 mg / kg. Twenty-four of the participants had valid renal ultrasounds at baseline and 11 had improvement in nephrocalcinosis grade from baseline and 4 remained stable. Only 3 children experienced a deterioration in class. In the 14 children with baseline nephrocalcinosis and available ultrasounds, 11 showed improvement from baseline and 8 of those children saw improvement in both kidneys. Common side effects included injection site reaction, erythema, pain, itching and swelling.

Alnylam Pharmaceuticals, Inc. indicated that more data would be available later this year.


1. Alnylam Pharmaceuticals, Inc. Alnylam Announces Positive Early Clinical Outcome Results of ILLUMINATE-A Phase 3 Study of OXLUMO® (lumasiran). Published May 3, 2021. Accessed May 3, 2021.

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