September 30, 2020
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Results show benefits of drug to treat primary hyperoxaluria type 1 in young children

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Phase 3 results from the Illuminate-B study suggest lumasiran, an investigational RNA interference therapeutic, may be safe and effective for treating pediatric patients who have primary hyperoxaluria type 1.

According to a press release from Alnylam Pharmaceuticals Inc., the study is the first to evaluate the safety and efficacy of RNA interference-based medications in children younger than 6 years of age, including infants.

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In the press release, Pritesh J. Gandhi, PharmD, vice president and general manager of the lumasiran program at Alnylam, noted the results from Illuminate-B are similar to those of Illuminate-A, which showed the medication was effective in reducing the hepatic production of oxalate in children 6 years and older.

“The current standard of care for young children and infants diagnosed with [primary hyperoxaluria type 1] PH1 is burdensome, including the frequent need for gastrostomy tube placement to enable hyperhydration, and, for those who have progressed to advanced disease, the risks associated with performing dialysis and, ultimately, organ transplantation,” he said. “Thus, we believe, a meaningful reduction in urinary oxalate levels has the potential to favorably impact disease progression and management in very young patients.”

For the Illuminate-B study, researchers enrolled 18 patients with the condition at nine study sites across five countries. Patients were aged between 3 and 72 months and had an eGFR greater than 45/mL/min/1.732 (or normal serum creatinine for patients younger than 12 months).

At 6 months, researchers observed treatment with lumasiran resulted in a reduction of urinary oxalate across various weight categories, while also demonstrating beneficial impacts on urinary and plasma oxalate. No severe adverse events occurred.

Kim Hollander, executive director of the Oxalosis and Hyperoxaluria Foundation, also commented on the results.

“The ILLUMINATE-B results signal hope for the many families with children whose lives are deeply impacted by PH1,” she said in the release. “This is especially encouraging given that children as young as a few months old could benefit from the therapeutic approach that lumasiran offers, curbing production of oxalate at its source.”

The company anticipates reporting full study results at the American Society of Nephrology’s Annual Meeting, which will take place virtually at the end of October.