ILLUMINATE-C phase III open-label study of Oxlumo shows efficacy in primary hyperoxaluria type 1.
Results of the primary analysis at six months demonstrated substantial reduction in plasma oxalate from baseline in patients (N=21) with advanced disease, including those on hemodialysis. Elevated plasma oxalate is directly related to the pathophysiology of oxalosis and results in systemic deposition of oxalate in extra-renal tissues, potentially leading to bone fractures, cardiomyopathy, impaired erythropoiesis, vision loss, skin ulcers, and other serious manifestations1. The safety and tolerability profile of lumasiran following six months of treatment is encouraging across all ages, with no drug related serious adverse events (SAEs) and injection site reactions (ISRs) as the most common adverse event (AE).
Based on these results, the Company plans to submit a Supplemental New Drug Application (sNDA) for lumasiran with the U.S. Food and Drug Administration (FDA) and a Type II Variation with the European Medicines Agency (EMA) in late 2021.
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