CHOLBAM® (Cholic Acid) Now in Lecheng for Rare Bile Acid Disorder

Recently, the innovative drug CHOLBAM® (cholic acid capsules) has become accessible in the Boao Lecheng International Medical Tourism Pilot Zone for the treatment of inborn errors of bile acid synthesis (IEBAS). This rare metabolic disorder, long lacking targeted therapies, now has a new treatment option available to patients in China without the need to travel abroad.

Inborn errors of bile acid synthesis are a group of rare metabolic diseases caused by genetic defects in the enzymes involved in bile acid synthesis. Bile acids play an essential role in maintaining the enterohepatic circulation and promoting the absorption of fats and fat-soluble vitamins. When enzyme defects occur, normal bile acid production is blocked, leading to the accumulation of abnormal bile acids and toxic metabolic intermediates, which can impair liver function and systemic metabolism.

Clinical manifestations include cholestasis, abnormal liver function, fat and fat-soluble vitamin malabsorption, and in some patients, rickets and growth impairment. Because the clinical presentation varies depending on the specific enzyme defect, diagnosis and recognition remain challenging.

IEBAS has been included in China’s First List of Rare Diseases, jointly issued by the National Health Commission and four other government departments. However, due to the rarity of the condition and limited clinical awareness, many patients have historically relied on symptomatic supportive care rather than etiological treatment. Symptomatic management cannot correct the underlying defects in the bile acid synthesis pathway, leaving disease control inadequate and long-term prognosis poor.

CHOLBAM® (cholic acid capsules) is a bile acid replacement therapy approved by the U.S. Food and Drug Administration for the treatment of bile acid synthesis disorders due to single enzyme defects. It is also indicated as adjunctive therapy for patients with peroxisomal disorders—including Zellweger spectrum disorders—who present with liver disease manifestations, steatorrhea, or complications of fat-soluble vitamin malabsorption.

The therapeutic principle is to supplement exogenous cholic acid, which participates in the enterohepatic circulation and exerts feedback regulation on the bile acid synthesis pathway. This reduces the formation of abnormal bile acids and toxic intermediate metabolites, thereby improving bile acid metabolism. Treatment has been shown to improve cholestasis and liver function, promote fat and fat-soluble vitamin absorption, and may positively influence growth parameters in some patients.

Leveraging the unique “pioneering pilot” policy of the Boao Lecheng Pilot Zone, CHOLBAM® has been introduced to China ahead of full national approval. This allows domestic IEBAS patients to access this globally advanced therapy close to home, without the need for overseas travel.

In a further step to improve affordability and sustainability of long-term treatment, CHOLBAM® has been included in the Lecheng Global Special Drug Insurance 2026 edition. This inclusion provides financial support for patients requiring innovative therapies and helps alleviate the economic burden associated with lifelong disease management. For rare disease patients who require continuous and long-term care, this makes innovative treatment more accessible and sustainable.