First treatment for rare condition primary hyperoxaluria type 1

Photo by Jakayla Toney

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.

Oxlumo will be available as solution for injection (189 mg/ml). The active substance of Oxlumo is lumasiran, a small interfering ribonucleic acid that causes degradation of the messenger ribonucleic acid involved in the synthesis of the enzyme glycolate oxidase (GO) in the liver, leading to decreased GO enzyme levels in the body. This results in reduction of plasma and urinary oxalate levels, the underlying cause for the symptoms in patients with PH1.

The benefit of Oxlumo is its ability to reduce oxalate levels in the plasma and 24-hour urinary oxalate excretion, when compared to a placebo treatment. This was observed during a 6-month clinical trial involving 38 patients aged 6-60. In this study, treatment with Oxlumo resulted in normalisation of oxalate excretion in 52% of patients and near normalisation in 84% of patients. The most common side effects are injection site reactions and abdominal pain.

Oxlumo was accepted in EMA’s PRIME scheme and has benefited from the extra support offered by the Agency to medicines that have a particular potential to address patients' unmet medical needs. EMA’s human medicines committee (CHMP) reviewed the application for Oxlumo under its accelerated assessment procedure, which allows the speeding up of patients' access to medicines.

The opinion adopted by the CHMP is an intermediary step on Oxlumo’s path to patient access. The opinion will now be sent to the European Commission for the adoption of a decision on an EU-wide marketing authorisation. Once a marketing authorisation has been granted, decisions about price and reimbursement will take place at the level of each Member State, taking into account the potential role/use of this medicine in the context of the national health system of that country.

Notes: 

Original source European Medicines Agency

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