• Bylvay^® is the first once-daily ileal bile acid transport inhibitor to be approved as a treatment for pruritus associated with progressive familial intrahepatic cholestasis (PFIC) in Japan, offering a non-surgical treatment option for infants, young children and adults
• PFIC is a rare and life-threatening liver disease that affects an estimated 100 children and infants in Japan.

 Ipsen  announced  that Japan’s Ministry of Health, Labour and Welfare (MHLW) has granted regulatory approval for Bylvay^® (odevixibat) for the treatment of pruritus associated with progressive familial intrahepatic cholestasis (PFIC). PFIC is a group of rare genetic disorders in which bile acid accumulates in the liver, leading to progressive liver damage and potentially liver failure. The condition severely impacts quality of life through debilitating symptoms such as severe itching (pruritus), caused by the bile accumulation in the liver and bloodstream, which can cause skin mutilation, sleep disruption, irritability, and impaired cognitive and social development.

“Children with PFIC often endure relentless itching that affects their daily quality of life. This includes regular, nightly sleep disturbance, which can have a negative impact on the whole family,” said Sandra Silvestri, MD, PhD, Executive Vice President, Chief Medical Officer, Ipsen. “The approval of Bylvay as a once-daily oral therapy represents a welcomed new option in how we approach this disease and offers new hope for patients and families in Japan living with the devastating impact of PFIC.”

Bylvay is a potent, once-daily, oral, ileal bile acid transport inhibitor (IBATi) that reduces reabsorption of bile acid back to the liver. In PEDFIC, the largest global Phase III trial conducted in PFIC, children treated with Bylvay showed significant improvements in serum bile acid and pruritus severity. The treatment was generally well-tolerated, with no drug-related serious adverse events and a low incidence of gastrointestinal events.

“Early diagnosis and intervention are critical in PFIC to manage symptoms and preserve liver function,” said Dr. Hiroki Kondou, Associate Professor, Department of Pediatrics, Kindai University Nara Hospital. “This approval of Bylvay provides patients and caregivers with a new treatment option that has the potential to reduce itching, and thereby improve sleep quality, while potentially supporting the preservation of liver.”

The approval from the MHLW was based on data from a Phase III, open-label study conducted in Japan, which evaluated the efficacy and safety of odevixibat in pediatric patients with PFIC types 1 and 2. The study confirmed improvements in serum bile acid levels and pruritus consistent with the global Phase III PEDFIC results. The Phase III clinical development plan for Bylvay in Japan was managed by Jadeite Medicines Inc and as part of the strategic collaboration, the new drug application for this indication was transferred to Ipsen headquartered in Tokyo, Japan. Ipsen will also be responsible for commercialization of Bylvay in Japan.