Pharmaceutical Business review

Japan MHLW grants orphan drug status to Renalys’ sparsentan for IgAN treatment

Sparsentan is intended to treat primary IgA nephropathy, a prominent cause of kidney failure. Credit: Robina Weermeijer on Unsplash.

This designation is effective as of 27 November this year.

Sparsentan was originally developed by Travere Therapeutics and was in-licensed to Renalys Pharma, which holds license for its development and commercialisation in Japan and several other Asian countries.

The therapy received full Food and Drug Administration (FDA) approval in the US under the brand name FILSPARI in September this year, to slow kidney function decline in adult people with primary IgAN and having a progression risk of the disease.

Renalys Pharma chief development officer Ryutaro Shimazaki said: “IgA nephropathy, a leading cause of kidney failure, occurs when abnormal IgA proteins deposit in kidney tissue, leading to inhibited kidney function and inflammation. There is an extremely high unmet medical need in Japan, as no approved drugs are currently indicated for this disease.

“We have in-licensed the Japan and Asian rights to sparsentan, which has already received approval in Europe and the US and are advancing clinical development leveraging the global phase III PROTECT data in order to address the kidney function loss in this disease.”

Renalys Pharma plans to bring the therapy to the Japanese market as swiftly as possible to address unmet medical needs in kidney disease.

IgA nephropathy, a prominent cause of kidney failure, occurs when abnormal IgA proteins accumulate in the kidneys, leading to inflammation and impaired kidney function.

As a rare and difficult-to-treat disease, there is a substantial need for effective treatments for IgA nephropathy in Japan.

Founded in 2023 by Catalys Pacific and SR One, Renalys Pharma is a Japan-based late-stage clinical biopharmaceutical company.