Selective PPARα Modulator "K-808" (Pemafibrate) Granted "Breakthrough Therapy" Designation by the U.S. Food and Drug Administration (FDA) As a Treatment for Primary Biliary Cholangitis

NAGOYA, Japan, June 30, 2026 /PRNewswire/ -- Kowa Company, Ltd. (Headquarters: Nagoya, Aichi Prefecture, Japan; hereinafter "Kowa"), today announced that on June 11, it received Breakthrough Therapy designation from the U.S. Food and Drug Administration (FDA) for "K-808" (development code, generic name: pemafibrate) for the treatment of patients with primary biliary cholangitis (PBC).

Breakthrough Therapy designation is a program granted by the FDA to expedite the development and review of drugs for which preliminary data or evidence suggests a substantial improvement over existing treatments in one or more clinically important endpoints for serious diseases.

K-808 was designated as a breakthrough therapy based on preliminary data and evidence from the ongoing Phase II clinical trial (K-808-2.01). The results of this trial suggest a significant improvement over existing treatments, as measured by a reduction in alkaline phosphatase (ALP) levels. Preliminary data from the trial were presented at the European Association for the Study of the Liver (EASL) Congress held in May 2026.

Kowa is advancing the development of "K-808" with the aim of obtaining global approval, including in the United States and Japan, for the treatment of PBC in combination with ursodeoxycholic acid (UDCA) in adult patients who have an inadequate response to UDCA, or as monotherapy in patients unable to tolerate UDCA.

 Through the development of "K-808," Kowa aims to provide new treatment options to a greater number of PBC patients.

• What is Pemafibrate?
  
  Pemafibrate is a selective PPARα modulator (Selective Peroxisome Proliferator-Activated Receptor α Modulator: SPPARMα) developed by Kowa. It comprehensively improves lipid metabolism, such as by lowering blood triglyceride (TG) levels, primarily by regulating the expression of target genes involved in lipid and glucose metabolism in the liver. In Japan, "Parmodia Tablets" were launched on June 1, 2018, for the treatment of hyperlipidemia.
  
  
• Development of K-808 (Development of Pemafibrate for PBC)
  
  Kowa is currently developing a therapeutic agent using pemafibrate aimed at delaying disease progression in patients with PBC. In the treatment of PBC, the drug is expected to take effect through mechanisms of action such as micellization of hydrophobic bile acids, anti-inflammatory effects resulting from the activation of the nuclear receptor PPARα, and inhibition of bile acid synthesis. 
  
  
• Primary Biliary Cholangitis (PBC)
  
  PBC is a chronic, progressive cholestatic liver disease and a severe, life-threatening autoimmune liver disease. As Inflammation and destruction of the intrahepatic bile ducts progress, it may potentially result in fibrosis, cirrhosis, and liver failure. Other clinical symptoms include fatigue and pruritus (itching), which can be extremely severe in some patients. PBC is a rare disease that primarily affects women.

Media Contact:
Ian Mehr
Kowa Research Institute, Inc.
919-433-1600
imehr@kowaus.com 

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