Alnylam's Oxlumo backed by NICE as first therapy to treat underlying cause of PH1

The UK’s National Institute for Health and Care Excellence (NICE) has issued a final decision recommending the use of Oxlumo (lumasiran) on the National Health Service (NHS) in England as an option for treating primary hyperoxaluria type 1 (PH1) in people of all ages, US RNAi therapy specialist Alnylam Pharmaceuticals (Nasdaq: ALNY) announced today.

Alnylam notes that lumasiran is the first treatment made available in England that has the potential to lower harmful oxalate levels, in the majority of patients, that drive the progression of PH1. Without treatment, people living with this disease can experience recurrent kidney stones from a very young age and potentially develop progressive kidney failure and end-stage kidney disease, following which treatment options are often limited to dual or sequential liver-kidney transplants. The drug has regulatory approval in major markets, including the UK, USA and European Union.

PH1 is an ultra-rare, inherited disease caused by the overproduction of a substance called oxalate. In this disease, a genetic alteration causes the liver to produce excess oxalate which can then form toxic crystals that can damage various parts of the body. Around 120 people in the UK have primary hyperoxaluria and about 80% of cases are diagnosed as type 1 – the most severe and common form of primary hyperoxaluria.