FDA approves first treatment for primary hyperoxaluria type 1
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The FDA has approved Alnylam Pharmaceuticals Inc.’s lumasiran, known as Oxlumo, for the treatment of primary hyperoxaluria type 1 in both adults and children.
The RNA interference therapeutic, also approved by the European Union last week, is the first medication approved to treat the condition and is in the form of a subcutaneous injection, according to a company press release.
Healio Nephrology previously reported on phase 3 results of the ILLUMINATE-B study, which demonstrated the safety and efficacy of the drug for lowering oxalate levels in pediatric patients (the ILLUMINATE-A study had similar results, but involved patients 6 years of age and older).
According to Jeffrey M. Saland, MD, professor and chief of pediatric nephrology and hypertension at Mount Sinai Kravis Children’s Hospital and an investigator on the ILLUMINATE-A trial, patients with primary hyperoxaluria type 1 (PH1) experience progressive decline in kidney function, with the risk of end-stage kidney disease increasing as the disease advances.
“This condition, systemic oxalosis, leads to multi-organ dysfunction and death,” he said in the release. “The age of onset, rate of disease progression and associated clinical manifestations can vary significantly from patient to patient, even among members of the same family, making PH1 a particularly challenging condition to diagnose and treat. Until today, there had been no approved nonsurgical treatment options available that curb oxalate overproduction in patients with PH1, with liver transplantation being the only preemptive treatment approach to address the underlying metabolic defect in these patients.”
Kim Hollander, executive director of the Oxalosis and Hyperoxaluria Foundation, also commented on the approval in the release.
“Many people impacted by PH1 face persistent anxiety related to the unpredictable nature of their condition, in terms of the uncertainty of how quickly their disease may progress, and the prospect of needing intensive dialysis and a kidney/liver transplant that threaten their physical, emotional and financial health,” she said. “The FDA approval of Oxlumo represents a new path forward for many, providing an effective treatment option and a sense of hope.”
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