Jadeite Medicines Secures Orphan Drug Designation for Odevixibat to Treat Alagille Syndrome
Thursday, December 26, 2024
Jadeite Medicines has announced that odevixibat has been granted Orphan Drug Designation by Japan’s Ministry of Health, Labour and Welfare (MHLW) for the treatment of Alagille Syndrome (ALGS).
ALGS is a rare genetic disorder that affects multiple organ systems, including the liver, heart, skeleton, eyes, and kidneys. The condition is caused by abnormalities in bile ducts, leading to bile acid build-up, liver scarring, and progressive liver disease.
Most individuals with ALGS experience chronic cholestasis, with symptoms often appearing in infancy. Severe, persistent itching affects up to 88% of patients. The global incidence of ALGS is estimated at 3 per 100,000 live births.
In Japan, ALGS is recognised as an intractable disease (designation number 297) by the MHLW, with an estimated 200–300 affected individuals nationwide.
Odevixibat is an established treatment for cholestatic pruritus in ALGS and progressive familial intrahepatic cholestasis (PFIC) in the United States and European countries, where it is marketed by Ipsen.
The drug is a once-daily, non-systemic ileal bile acid transport inhibitor (IBATi) that works locally within the small intestine.
In addition to its approval for ALGS and PFIC, odevixibat has received orphan drug designations for biliary atresia and primary biliary cirrhosis in the United States and Europe.
The Phase III BOLD trial for biliary atresia is ongoing internationally.
Jadeite Medicines continues its efforts to advance drug development to address unmet medical needs and improve the lives of patients with rare diseases.
Source: jadeitemedicines.co.jp
