Major Breakthrough: Rare Disease Innovative Drug BYLVAY (Odevixibat Capsules) Officially Launched in China

　　【Pharmaceutical Network Product Information] Recently, there is good news in the field of rare disease drugs! It is reported that Ipsen announced the official commercial launch of its innovative rare disease drug BYLVAY, Odevixibat capsules (Chinese name: Beierwei, Odevixibat capsules) in China. This marks a significant breakthrough in the availability of treatment drugs for Progressive Familial Intrahepatic Cholestasis (PFIC).
 

Data show that Progressive Familial Intrahepatic Cholestasis (PFIC) is an autosomal recessive genetic disorder caused by mutations in genes such as ABCB11 and MYO5B, leading to bile metabolism disorders. It is clinically characterized by severe pruritus and low GGT cholestasis, commonly occurring in infants and young children, and can progress to liver failure. The main manifestations include jaundice, hepatosplenomegaly, and rickets during infancy.
 

International data shows that the overall incidence of PFIC is approximately (1-2)/100,000, encompassing 13 different subtypes, making it "rare among rare diseases." Although China has not yet conducted precise incidence calculations, based on the population base, the estimated number of existing patients in China should be over a thousand.
 

Odevixibat capsules were officially approved by the China National Medical Products Administration in December 2024 for the treatment of pruritus in PFIC patients aged ≥6 months. As a first-line treatment for PFIC, Odevixibat is the first non-surgical targeted drug approved in China for the treatment of PFIC, providing patients with an effective and safe non-invasive drug treatment option.
 

Data show that, as a reversible, potent, and selective inhibitor of the ileal bile acid transporter (IBAT), this drug exerts its therapeutic effects through a unique mechanism of action—by inhibiting the reuptake of bile acids in the distal ileum, increasing bile acid clearance through the colon, thereby reducing serum bile acid concentration and alleviating pruritus symptoms in patients.
 

An industry insider said that drugs used to block the enterohepatic circulation in the past needed to physically bind with bile acids to take effect, thus requiring multiple doses per day and administration before meals. However, as a receptor antagonist, Odevixibat typically requires only once-daily dosing, greatly improving patient medication adherence. The drug acts locally in the intestine with minimal systemic exposure, offering higher safety.
 

The person stated that PFIC is a type of rapidly progressing rare pediatric liver disease, which can lead to liver fibrosis, cirrhosis, and end-stage liver complications, often requiring a liver transplant for long-term survival. In some subtypes of PFIC, only half of the children can survive with their native liver until the age of 10. Due to the rarity of the disease and its complex clinical manifestations, diagnosis and treatment face numerous challenges. As an innovative ileal bile acid transporter (IBAT) inhibitor, Odevixibat provides a new non-invasive drug treatment option for PFIC patients, helping to reduce serum bile acids and alleviate pruritus symptoms, with the potential to improve long-term native liver survival and quality of life for children with PFIC.
 

Data shows that Ipsen is a global biopharmaceutical company focused on providing innovative drugs to patients in three therapeutic areas: oncology, rare diseases, and neuroscience. Ipsen entered China in 1992, established its China headquarters in Shanghai in 2021, and divested its diversified healthcare business in 2022 in line with the group’s strategic adjustments to focus on specialty medicines. Targeting the three major disease areas (oncology, rare diseases, and neuroscience), Ipsen China will continue to introduce innovative treatment solutions to meet the urgent unmet medical needs of Chinese patients.
 

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