Jeroen Valkenburg, General Manager of the Lumasiran program at Alnylam Pharmaceuticals, discusses the reaction of the primary hyperoxaluria type 1 (PH1) community following the approval of lumasiran last November.
PH1 is a rare genetic condition that is often difficult to diagnose since its main symptom is the development of kidney stones. These rare type of kidney stones are formed due to the build up of oxalate, which normally is filtered through the kidneys and excreted. PH1 is due to mutations in the AGXT gene.
As Mr. Valkenburg notes, everyone in the PH1 community is excited about the availability of lumasiran. Feedback from physicians, caregivers, and patients has been positive as lumasiran now gives these patients the ability to lower urinary oxalate, which leads to improved long-term clinical outcomes.
The FDA approval of lumasiran was based on the results of the ILLUMINATE-A and ILLUMINATE-B clinical trials, which demonstrated the drug’s safety and efficacy in lowering urinary oxalate levels in pediatric and adult patients. Recently, at Kidney Week 2021, 6-month primary analysis data from the ILLUMINATE-C trial of lumasiran was presented which is evaluating the ability of lumasiran to reduce plasma oxalate in PH1 patients with chronic kidney disease stage 3b-5 with or without dialysis. As Mr. Valkenburg explains, plasma oxalate is incredibly dangerous as it is associated with systemic oxalosis, which can cause cardiomyopathy, blindness, skin ulcers, and bone fractures. The 6-month primary analysis data demonstrate that treatment with lumasiran resulted in substantial reductions in plasma oxalate and an acceptable safety profile through Month 6 in this subset of PH1 patients with chronic kidney disease stage 3b-5.
For more information about PH1 and other kidney disorders, click here.
