NICE recommends OXLUMO®▼ (lumasiran) in England as first therapy to treat underlying cause of ultra-rare condition primary hyperoxaluria type 1 (PH1)
pharmafile | March 20, 2023 | News story | Business Services |
MAIDENHEAD, UK, Monday 20th March 2023 – Alnylam UK Limited, the leading RNA interference (RNAi) therapeutics company, today welcomed a final decision from the National Institute for Health and Care Excellence (NICE) recommending the use of OXLUMO® (lumasiran) on the NHS in England as an option for treating primary hyperoxaluria type 1 (PH1) in people of all ages.[i] 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.
Professor Sally-Anne Hulton, Consultant Paediatric Nephrologist, Birmingham Women’s and Children’s Hospital NHS Trust, UK said: “Until recently, there have been very limited options for people affected by PH1. For most patients with PH, management would focus on reducing symptoms and slowing kidney damage without addressing the underlying cause. Today’s decision changes that. We will be able to offer patients a therapy that treats the root cause of the disease acting directly to reduce toxic oxalate levels.”
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 approximately 80% of cases are diagnosed as type 1 – the most severe and common form of primary hyperoxaluria. [ii],[iii]
Helen Morris, Individual Support Lead, Metabolic Support UK, said: PH1 is a serious condition that can affect people of all ages. It can be particularly devastating in infants and young children who generally have the most severe disease outcomes and whose kidney function can deteriorate rapidly, resulting in urgent hospital admission. Today’s announcement is a welcome advance in the treatment of this disease that has life-long physical and emotional implications for patients, their families, and carers. This decision will provide much-needed reassurance for patients and their families that an effective treatment option to manage their condition is available.”
Lumasiran is an RNA-interference (RNAi) therapeutic that silences a gene called HAO1 and in turn, reduces production of an enzyme called glycolate oxidase (GO). GO is responsible for producing oxalate, a toxic product that is normally excreted in the urine.[iv] In people with PH1 however, an excess amount of oxalate is produced that accumulates in the kidneys and can cause kidney stones and progressive damage. If the damage is severe, the kidneys may fail and this is when oxalate can begin to deposit in other organs and cause further harm, including irreversible damage to the eyes, bones, skin and heart.
Philip Davey, Country Manager of Alnylam Pharmaceuticals, UK and Ireland said: “We are committed to ensuring that the innovative RNAi therapeutics we develop for serious diseases can be accessed by patients who need them. No matter how many people a disease affects – whether hundreds, thousands or millions – it is our mission to ensure no patient who could benefit from treatment is left behind. Today’s decision is therefore a very welcome milestone for the PH1 community in England. We hope that the availability of lumasiran in England will help patients and families living with this potentially devastating disease.”
[i] NICE. Lumasiran for treating primary hyperoxaluria type 1 [ID3765]. Last accessed: February 2023.
[ii] Research – Rare Renal. The UK Kidney Association. Available at: https://ukkidney.org/rare-renal/research-rare-renal. Last accessed: Last accessed: February 2023.
[iii] Poyah et al., 2021. Primary Hyperoxaluria Type 1 (PH1) Presenting With End-Stage Kidney Disease and Cutaneous Manifestations in Adulthood: A Case Report. Canadian Journal of Kidney Health and Disease, 8, https://doi.org/10.1177/20543581211058931. Last accessed: Last accessed: February 2023.
[iv] Lumasiran Summary of Product Characteristics. Last accessed: February 2023.
