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This search function provides links to outputs produced by NIHR Innovation Observatory. These are briefing notes or reports on new or repurposed technologies. This search will not return all technologies currently in development as these outputs are produced as required for our stakeholders.

Innovation Observatory > Reports > Drugs > Lumasiran for primary hyperoxaluria type I

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Lumasiran for primary hyperoxaluria type I

Drugs

Renal Disease and Urology

April 2019


Lumasiran is in clinical development for the treatment of primary hyperoxaluria type I (PH1). PH1 is a very rare disease caused by certain genetic mutations, in which excess oxalate production results in the deposition of oxalate crystals in the kidneys and urinary tract. This leads to stone formation and kidney failure with significant morbidity and mortality. Treatment options for PH1 include vitamin B6 which is known to reduce the body’s production of oxalate, dietary recommendations to prevent kidney stones and combined liver-kidney transplantation before or after development of end-stage kidney failure.
Lumasiran, which is administered as a subcutaneous injection, is designed to reduce the levels of an enzyme called glycolate oxidase produced by the liver. Oxalate production is therefore inhibited. By reducing oxalate production, lumasiran has the potential to prevent the actual disease process that develops in PH1. If licensed, lumasiran may provide the first pharmacological treatment option for patients with PH1 who do not have any approved treatment.

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