The FDA announced the approval of Alnylam Pharmaceuticals' Oxlumo (lumasiran), making it the first treatment for primary hyperoxaluria type 1 (PH1) authorised in the US. The decision follows the EU approval of the RNAi therapeutic last week for use in the same indication.
The FDA approval is based on a priority review of data from two Phase III studies of Oxlumo in patients with PH1. The ILLUMINATE-A trial enrolled 39 adults and children six years or older who received either a monthly injection of Alnylam's therapy followed by a maintenance dose every three months, or placebo. The study met its primary endpoint, with Oxlumo resulting in an average 65% reduction in urine oxalate excretion, compared to an average 12% reduction for placebo. By the sixth month, 52% of patients treated with Oxlumo reached a normal 24-hour urinary oxalate level, while none in the placebo arm did.
Meanwhile, the FDA also took into consideration data from the single-arm ILLUMINATE-B study, which included 16 infants and children under the age of six. Results there showed that Oxlumo led to an average 71% decrease in urinary oxalate by month six.
As the cost for Oxlumo may vary given that it is dosed by weight and prescribed across a wide spectrum of ages, Alnylam said that it has structured a new value-based agreement (VBA) component that specifically addresses US payers' concerns regarding budget predictability and value. According to Alnylam, the so-called Patient Need Adjustment (PNA) component is "designed to mitigate the risk of escalating or varying costs associated with dosing requirements, thereby accelerating access for people diagnosed with PH1."
Further, in order to address the issue of budget predictability, Alnylam is also making available a Prevalence Based Adjustment (PBA) component, first introduced last year for its hepatic porphyria drug Givlaari (givosiran). "This feature triggers a rebate to participating payers if the number of diagnosed patients they cover exceeds current epidemiologic estimates for PH1," the company explained, adding that Express Scripts, Harvard Pilgrim, and Highmark are among the payers pursuing VBAs for Oxlumo.
Andy Orth, who heads the US region for Alnylam, said the PNA "was born out of direct feedback from our payer partners, and experience with our first two approved therapies." The executive added that the company "will continue to pursue commercial innovation that offers good value and straightforward pricing for potentially transformative RNAi therapies like Oxlumo." The drug is expected to be available for shipment to healthcare providers in the US by year-end.
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