EMA has recommended granting a marketing authorisation in the European Union for Oxlumo (lumasiran) for the treatment of primary hyperoxaluria type 1 (PH1).
Primary hyperoxaluria is a rare inherited disorder characterised by the overproduction of oxalate. Oxalate can form calcium oxalate deposits, which can cause stones in the kidney and urinary tract (structures that carry urine) as well as injury to other organs such as the heart, eyes, bones and skin. Characteristic symptoms of the disease include renal colic, blood in the urine, frequent urinary tract infections and stomach pain.
PH1 is the most common and the most severe form of the disease, accounting for 80% of all cases. The condition is very rare with an estimated prevalence of 0.05 in 10,000 people in the EU.
There are currently no approved medicines for PH1 in the EU. Different treatments are used to prevent the accumulation of calcium oxalate such as dietary changes, drinking plenty of fluids and taking vitamin B6. In certain cases kidney and/or liver transplantation is required. Untreated PH1 leads to kidney failure, which is life-threatening.