Renalys Pharma, Inc., a biopharmaceutical company advancing in late-stage clinical trials, has announced that its drug sparsentan, acquired from Travere Therapeutics, Inc. (Travere), currently undergoing phase III studies in Japan, has been granted Orphan Drug Designation by Japan’s Ministry of Health, Labour and Welfare for primary IgA nephropathy as of November 27, 2024.
In Japan, drugs for rare diseases affecting fewer than 50,000 patients and marked by high unmet medical needs are designated by the Ministry of Health, Labour and Welfare as orphan drugs. This designation qualifies sparsentan for priority review, promising to expedite the marketing approval process by several months.
“IgA nephropathy, a primary cause of kidney failure, manifests through the deposition of abnormal IgA proteins in kidney tissues, impairing their function and causing inflammation. The need for effective treatment in Japan is critical, as no existing medications have been approved for this condition,” stated Renalys Pharma’s Chief Development Officer, Ryutaro Shimazaki. “We have secured the rights to develop and market sparsentan in Japan and across Asia, building upon its approval in Europe and the USA. Leveraging global phase III PROTECT trial data, we aim to address kidney function deterioration for this disease. The orphan designation will accelerate our efforts in Japan, allowing us to potentially deliver this innovative therapy to patients sooner.”
Developed originally by Travere, sparsentan, for which Renalys Pharma holds exclusive development and commercialization rights, is set to reach markets across Japan, South Korea, Taiwan, and several Southeast Asian countries. In September 2024, Travere obtained full FDA approval for sparsentan (known commercially as Filspari in the US) to decelerate kidney impairment in adults with primary IgA nephropathy at risk of progression.
Renalys Pharma is committed to improving patient outcomes in Japan and Asia, focusing on groundbreaking kidney disease treatments, with sparsentan at the forefront of these efforts.
The management of chronic kidney disease (CKD) and end-stage renal disease (ESRD) poses significant challenges to the Japanese healthcare system. Widely recognized as a leading cause of kidney failure, IgA nephropathy is characterized by the deposition of abnormal IgA proteins in renal tissue, blocking functionality and leading to inflammation. Identified as a rare, complex illness (classified under intractable disease 66), it demands innovative treatment solutions, currently unmet in Japan’s medical landscape.
Renalys Pharma, a private entity focused on the development of innovative renal therapies, remains dedicated to addressing unmet needs within renal disease management for Japanese and Asian populations.