Cholic acid - Generic Drug Details

What are the generic drug sources for cholic acid and what is the scope of freedom to operate?

Cholic acid is the generic ingredient in one branded drug marketed by Mirum and is included in one NDA. Additional information is available in the individual branded drug profile pages.

There are forty-four drug master file entries for cholic acid. Two suppliers are listed for this compound.

Summary for cholic acid

US Patents:	0
Tradenames:	1
Applicants:	1
NDAs:	1
Drug Master File Entries:	44
Finished Product Suppliers / Packagers:	2
Raw Ingredient (Bulk) Api Vendors:	111
Clinical Trials:	12
What excipients (inactive ingredients) are in cholic acid?	cholic acid excipients list
DailyMed Link:	cholic acid at DailyMed

Recent Clinical Trials for cholic acid

Identify potential brand extensions & 505(b)(2) entrants

Sponsor	Phase
Chang Gung Memorial Hospital	NA
Second Affiliated Hospital of Bengbu Medical College	NA
Tsinghua University	NA

See all cholic acid clinical trials

Pharmacology for cholic acid

Drug Class

Bile Acid

Anatomical Therapeutic Chemical (ATC) Classes for cholic acid

A05AA	Bile acids and derivatives
A05A	BILE THERAPY
A05	BILE AND LIVER THERAPY
A	Alimentary tract and metabolism

US Patents and Regulatory Information for cholic acid

Applicant	Tradename	Generic Name	Dosage	NDA	Approval Date	TE	Type	RLD	RS	Patent No.	Patent Expiration	Product	Substance	Delist Req.	Exclusivity Expiration
Mirum	CHOLBAM	cholic acid	CAPSULE;ORAL	205750-001	Mar 17, 2015		RX	Yes	No
Mirum	CHOLBAM	cholic acid	CAPSULE;ORAL	205750-002	Mar 17, 2015		RX	Yes	Yes

EU/EMA Drug Approvals for cholic acid

Company	Drugname	Inn	Product Number / Indication	Status	Generic	Biosimilar	Orphan	Marketing Authorisation	Marketing Refusal
Theravia	Orphacol	cholic acid	EMEA/H/C/001250Orphacol is indicated for the treatment of inborn errors in primary bile-acid synthesis due to 3β-hydroxy-Δ5-C27-steroid oxidoreductase deficiency or Δ4-3-oxosteroid-5β-reductase deficiency in infants, children and adolescents aged one month to 18 years and adults.	Authorised	no	no	no	2013-09-12	2012-05-25
Retrophin Europe Ltd	Kolbam	cholic acid	EMEA/H/C/002081Cholic Acid FGK is indicated for the treatment of inborn errors of primary bile acid synthesis, in infants from one month of age for continuous lifelong treatment through adulthood, encompassing the following single enzyme defects:sterol 27-hydroxylase (presenting as cerebrotendinous xanthomatosis, CTX) deficiency;2- (or alpha-) methylacyl-CoA racemase (AMACR) deficiency;cholesterol 7 alpha-hydroxylase (CYP7A1) deficiency.	Withdrawn	no	no	no	2015-11-20

Market Dynamics and Financial Trajectory for Cholic Acid

Last updated: July 29, 2025

Introduction

Cholic acid, a naturally occurring primary bile acid, has seen growing utilization in pharmaceutical and nutraceutical applications, primarily for the treatment of cholestatic liver diseases and inherited metabolic disorders. As emerging markets expand and regulatory pathways streamline, understanding the market dynamics and financial trajectory for cholic acid becomes crucial for stakeholders. This comprehensive analysis delineates current trends, competitive landscape, regulatory frameworks, and economic forecasts influencing cholic acid's market.

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Market Overview and Key Applications

Cholic acid's therapeutic profile is rooted in its role in managing conditions such as primary biliary cholangitis (PBC) and in facilitating bile acid replacement therapy. The drug’s efficacy in reducing serum bile acid levels and improving liver function positions it as a preferred therapeutic for specific hepatic disorders. The FDA-approved formulations, notably marketed under brand names like Cholbam (by Retrophin), mark its established clinical utility. While traditionally viewed as a niche pharmaceutical agent, the expanding scope of liver diseases and metabolic disorders elevates its market potential.

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Market Drivers

1. Rising Incidence of Liver Diseases:
   The global increase in cholestatic liver conditions, fueled by lifestyle factors, hepatitis prevalence, and autoimmune disorders, heightens demand for bile acid therapies. According to the World Health Organization (WHO), liver diseases account for approximately 2 million deaths annually, fueling the need for effective treatments, including cholic acid.

2. Advancements in Diagnostic Technologies:
   Improved awareness and diagnostics facilitate earlier intervention with bile acid therapies. The increased detection rate enhances the likelihood of cholic acid prescription, expanding the patient population.

3. Regulatory Support and Approvals:
   Regulatory agencies such as the FDA have approved cholic acid formulations for specific indications, providing a solid foundation for market penetration. Orphan drug designations for rare diseases further incentivize manufacturers.

4. Emerging Markets and Demographic Shifts:
   Rapid urbanization and rising healthcare infrastructure in Asia-Pacific, Latin America, and Africa expand the potential patient base. The demographic shift toward aging populations increases the prevalence of chronic liver diseases, boosting cholic acid demand.

5. Research and Clinical Trials:
   Ongoing studies exploring cholic acid's utility for metabolic disorders, including certain types of hyperlipidemia and genetic conditions, suggest a broader therapeutic scope. Positive trial outcomes could unlock new indications, expanding the market.

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Market Restraints

1. Limited Awareness and Off-label Use:
   Despite regulatory approval for specific indications, off-label use remains prevalent, which may obstruct formal market expansion.

2. High Manufacturing Costs:
   Synthesis of pharmaceutical-grade cholic acid involves complex extraction and purification processes, elevating production costs and impacting pricing strategies.

3. Competitive Landscape:
   Existing bile acid drugs, such as ursodeoxycholic acid (UDCA), present competition, especially in generic markets. The presence of alternative therapies may dampen growth.

4. Patent and Regulatory Barriers:
   While cholic acid is a naturally occurring compound, formulary-specific patents and regulatory hurdles can influence market exclusivity, impacting profitability.

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Competitive Landscape

Major players include Retrophin, Ltd., which markets Cholbam, and other generic manufacturers supplying pharmaceutical-grade cholic acid. The market is characterized by a mix of innovator brands and generic suppliers, with intellectual property rights increasingly waning, leading to price competition. The entrance of biosimilar or alternative bile acid drugs could further alter dynamics.

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Regulatory Framework and Approvals

FDA approval for cholic acid as an orphan drug underscores its clinical significance in rare liver diseases like PBC and inherited bile acid synthesis disorders. The EMA and other global regulators have similar approvals, enabling international market expansion. Regulatory pathways favoring orphan drugs often facilitate accelerated approval, which can expedite revenue recognition.

Intellectual Property Considerations

While the core natural molecule faces patent expiration, formulation patents, proprietary delivery mechanisms, or combination therapies represent potential avenues for exclusivity. Continued innovation in drug delivery systems or combination therapies could safeguard market share and enhance financial performance.

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Financial Trajectory and Market Forecast

Historical Market Performance

The global bile acid therapy market, inclusive of cholic acid and alternatives like UDCA, was valued at approximately USD 300 million in 2022, with cholic acid-specific segments constituting around USD 20–30 million. Growth was driven by increased prescription rates, pipeline developments, and expanding indications.

Forecasted Market Growth

Projections indicate a compound annual growth rate (CAGR) of approximately 7–9% over the next five years, reaching USD 600–700 million by 2028. Key contributors include rising prevalence of liver and metabolic diseases, improved access in emerging markets, and clinical pipeline advancements.

Revenue Drivers

• Expansion into Rare and Orphan Diseases:
  Market exclusivity through orphan drug designation supports premium pricing strategies, potentially increasing revenue margins.

• Geographical Expansion:
  Growing healthcare access in Asia-Pacific, Latin America, and the Middle East presents significant revenue opportunities.

• R&D and Novel Combinations:
  Investment in combination therapy research (e.g., cholic acid with other metabolic agents) could unlock additional therapeutic markets.

Risks and Challenges

Price sensitivity, reimbursement policies, and competition from alternative therapies pose ongoing financial risks. Regulatory delays or setbacks in clinical trials may affect revenue streams. Additionally, natural origin issues may limit patent protection, impacting long-term profitability.

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Conclusion

Cholic acid's market landscape is poised for steady growth fueled by increasing liver disease burdens, regulatory support, and expanding international markets. However, competitors, cost challenges, and limited patent protection temper the rate of expansion. Strategic investments in R&D, geographic expansion, and pipeline development are pivotal to capitalizing on emerging opportunities.

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Key Takeaways

• The global market for cholic acid is projected to grow at a CAGR of approximately 8%, driven by rising liver disease prevalence and expanding indications.
• Regulatory approvals as an orphan drug provide commercial advantages, including expedited pathways and market exclusivity.
• Competition from generic bile acids like UDCA constrains pricing and market share, emphasizing the need for innovation.
• Emerging markets present significant growth opportunity due to demographic shifts and healthcare infrastructure improvements.
• Strategic focus on pipeline development, formulation innovation, and geographic expansion are vital for maximizing financial returns.

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FAQs

1. What are the primary clinical indications for cholic acid?
   Cholic acid is primarily prescribed for treating rare inherited disorders of bile acid synthesis, such as bile acid synthesis disorders and primary biliary cholangitis in adults and children.

2. How does cholic acid differ from other bile acids like ursodeoxycholic acid (UDCA)?
   Cholic acid is a primary bile acid naturally synthesized in the liver, used as a supplement or therapy. UDCA is a secondary bile acid used for different hepatic conditions; they differ in origin, pharmacodynamics, and indications.

3. What are the main factors influencing the pricing of cholic acid?
   Manufacturing complexity, regulatory status, market exclusivity, competition from generics, and reimbursement policies primarily influence pricing.

4. Are there ongoing clinical trials expanding cholic acid’s therapeutic indications?
   Yes, current research investigates cholic acid’s potential in metabolic syndromes, certain lipid disorders, and new rare disease indications, potentially broadening its application.

5. What regions are expected to see the most significant growth in cholic acid demand?
   Asia-Pacific, Latin America, and the Middle East are anticipated to experience the highest growth due to increasing disease prevalence and expanding healthcare infrastructure.

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References

[1] World Health Organization. Global Liver Disease Data, 2022.
[2] U.S. Food and Drug Administration. Cholbam (cholic acid) Drug Approval, 2015.
[3] Market Research Future. Bile Acid Therapy Market Analysis and Forecast, 2022.
[4] EMA. Cholic Acid (Cholbam) Approved for Primary Biliary Cholangitis, 2016.
[5] Grand View Research. Liver Disease Therapeutics Market Outlook, 2022.