原发性胆汁性胆管炎（PBC）是一种慢性疾病，会阻碍胆汁从肝脏流出，导致胆汁酸浓度升高，从而造成细胞损伤。PBC 如不及时治疗，可导致肝功能衰竭和死亡。在项目开始时，唯一获批的 PBC 治疗方法并非对所有患者都有效。
PK 模型支持肝功能受损患者的用药策略
PK 模型支持肝功能受损患者的用药策略
原发性胆汁性胆管炎（PBC）是一种慢性疾病，会阻碍胆汁从肝脏流出，导致胆汁酸浓度升高，从而造成细胞损伤。PBC 如不及时治疗，可导致肝功能衰竭和死亡。在项目开始时，唯一获批的 PBC 治疗方法并非对所有患者都有效。
Intercept Pharmaceuticals was developing obeticholic acid (OCA) as an alternative treatment for PBC. OCA activates farnesoid X receptors (FXR) in the liver to decrease bile acid concentration.
As liver damage occurs as PBC progresses, Intercept needed to develop an OCA dosing strategy for patients both with and without hepatic impairment. A clinical hepatic impairment study revealed that while systemic OCA concentrations increased as hepatic impairment worsened, the level of FXR activation in the liver remained similar across all groups. Plasma OCA concentration proved to be a poor surrogate for liver OCA concentration.
Intercept Pharmaceuticals was developing obeticholic acid (OCA) as an alternative treatment for PBC. OCA activates farnesoid X receptors (FXR) in the liver to decrease bile acid concentration.
As liver damage occurs as PBC progresses, Intercept needed to develop an OCA dosing strategy for patients both with and without hepatic impairment. A clinical hepatic impairment study revealed that while systemic OCA concentrations increased as hepatic impairment worsened, the level of FXR activation in the liver remained similar across all groups. Plasma OCA concentration proved to be a poor surrogate for liver OCA concentration.
These modeling results supported the safety and efficacy of OCA and the need to reduce dosing only for PBC patients with moderate and severe hepatic impairment.
Headquartered in New York, Intercept Pharmaceuticals is a biopharmaceutical company focused on the development and commercialization of novel therapeutics to treat progressive non-viral liver diseases, including PBC and nonalcoholic steatohepatitis (NASH). Founded in 2002, Intercept has operations in the United States, Europe, and Canada.
Headquartered in New York, Intercept Pharmaceuticals is a biopharmaceutical company focused on the development and commercialization of novel therapeutics to treat progressive non-viral liver diseases, including PBC and nonalcoholic steatohepatitis (NASH). Founded in 2002, Intercept has operations in the United States, Europe, and Canada.
