CHOLBAM Drug Patent Profile

When do Cholbam patents expire, and when can generic versions of Cholbam launch?

Cholbam is a drug marketed by Mirum and is included in one NDA.

The generic ingredient in CHOLBAM is cholic acid. There is one drug master file entry for this compound. One supplier is listed for this compound. Additional details are available on the cholic acid profile page.

Summary for CHOLBAM

US Patents:	0
Applicants:	1
NDAs:	1
Finished Product Suppliers / Packagers:	1
Raw Ingredient (Bulk) Api Vendors:	111
Clinical Trials:	1
Drug Prices:	Drug price information for CHOLBAM
What excipients (inactive ingredients) are in CHOLBAM?	CHOLBAM excipients list
DailyMed Link:	CHOLBAM at DailyMed

See drug prices for CHOLBAM

Recent Clinical Trials for CHOLBAM

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

Sponsor	Phase
University of Nebraska	Phase 1/Phase 2
Children's Hospital Medical Center, Cincinnati	Phase 1/Phase 2
University of Pittsburgh	Phase 1/Phase 2

See all CHOLBAM clinical trials

Pharmacology for CHOLBAM

Drug Class

Bile Acid

US Patents and Regulatory Information for CHOLBAM

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 CHOLBAM

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	Withdrawn	no	no	no	2015-11-20

Cholbam Market Dynamics and Financial Trajectory: Sales, Exclusivity, Competition, and Generic Risk

Last updated: August 25, 2026

Cholbam is a niche orphan drug with a defensible but limited commercial profile. Its active ingredient, cholic acid, is approved for bile acid synthesis disorders caused by single-enzyme defects and for adjunctive treatment of peroxisomal disorders, including Zellweger spectrum disorders. The product has benefited from orphan-drug pricing, limited physician familiarity with the disease area, and high switching costs. Its long-term growth is constrained by a very small patient population, diagnosis rates, reimbursement scrutiny, and the absence of broad-label expansion.

Travere Therapeutics has historically marketed Cholbam alongside Chenodal and other specialty products. Cholbam revenue has remained immaterial relative to Travere’s principal products, but the drug has strategic value because treatment is chronic, competition is limited, and manufacturing and regulatory requirements restrict rapid generic substitution.

What is Cholbam and which patients does it treat?

Cholbam is an oral cholic acid product supplied as capsules and granules. The FDA approved it in March 2015 under the orphan-drug pathway for:

• Treatment of bile acid synthesis disorders caused by single-enzyme defects.
• Adjunctive treatment of peroxisomal disorders, including Zellweger spectrum disorders, to improve abnormal bile acid production and support nutritional management.

Cholic acid replaces or supplements deficient primary bile acid. Treatment can reduce the accumulation of abnormal bile acid intermediates and improve bile flow and fat-soluble vitamin absorption in selected patients. The drug is not a general treatment for liver disease or cholestasis.

FDA regulatory profile

Attribute	Cholbam
Active ingredient	Cholic acid
Dosage forms	Capsules and oral granules
FDA application	NDA 205750
Approval date	March 17, 2015
Therapeutic category	Bile acid synthesis disorders and peroxisomal disorders
Regulatory pathway	Orphan drug and priority review framework
Sponsor at approval	Asklepion Pharmaceuticals
Historical commercial owner	Retrophin, later Travere Therapeutics
Patient population	Ultra-rare inherited metabolic and peroxisomal disorders

The label requires biochemical and clinical monitoring. Liver-function abnormalities and worsening hepatic disease can limit use. Pediatric administration is supported by the granule formulation, which broadens practical use in infants and young children.[1]

How large is the Cholbam market?

The addressable market is small in patient count but high in annual treatment value. The commercial market is determined by four variables: diagnosed patients, treatment persistence, dose by body weight, and payer approval.

Published epidemiology for the specific enzyme defects covered by the label is fragmented. Individual disorders such as 3-beta-hydroxy-delta-5-C27-steroid dehydrogenase deficiency, delta-4-3-oxosteroid-5-beta-reductase deficiency, and Zellweger spectrum disorders are rare. The total treated population is therefore likely measured in hundreds rather than tens of thousands in the United States and other high-income markets.

Market drivers

Cholbam demand is supported by several structural factors:

1. Chronic treatment. Patients who respond generally require long-term therapy.
2. Limited alternatives. There is no widely interchangeable, FDA-approved small-molecule competitor for the same labeled use.
3. Specialist prescribing. Treatment is concentrated among metabolic-disease, hepatology, gastroenterology, and pediatric specialists.
4. High clinical switching costs. Dose titration and monitoring make uncontrolled substitution unattractive.
5. Orphan-drug economics. A small population can support premium pricing if reimbursement is maintained.

Market constraints

Growth is restricted by the underlying disease biology and commercial infrastructure:

• Underdiagnosis delays initiation.
• Genetic and biochemical confirmation is required in many cases.
• The per-patient dose varies substantially.
• The product is dependent on specialty pharmacy and prior-authorization processes.
• Mortality and disease progression in peroxisomal disorders limit duration of treatment for some patients.
• Payers can require evidence that the patient meets the narrow FDA indication.
• International access is uneven because approval and reimbursement differ by country.

What has been Cholbam’s financial trajectory?

Cholbam has been a small, relatively stable revenue contributor rather than a primary growth engine for its commercial owner. Travere’s public filings historically grouped Cholbam with its rare-disease portfolio or disclosed it separately from larger products. Reported sales have generally been in the low tens of millions of dollars annually, with changes driven more by patient count, pricing, and product mix than by broad market expansion.[2][3]

Period	Financial pattern	Main interpretation
2015-2017	Commercial launch and patient identification	Revenue ramp depended on diagnosis and reimbursement
2018-2020	Low-double-digit-million-dollar annual contribution	Base of chronic patients developed
2021-2022	Stable specialty-product revenue with periodic fluctuations	Pricing and persistence offset limited patient growth
2023-2024	Mature, non-core product profile	Strategic value remained greater than absolute revenue scale

The financial model has three characteristics:

• High gross margin potential. Cholic acid is a small molecule with no complex biologic manufacturing process.
• High selling expense per patient. A field organization, medical affairs team, reimbursement support, and specialty distribution network must be maintained for a very small population.
• Low volume leverage. Incremental patient growth can materially affect revenue, but fixed commercial and regulatory costs limit operating leverage.

Cholbam’s financial trajectory is therefore more likely to show modest price-supported growth, flat revenue, or gradual erosion than rapid expansion. A meaningful inflection would require improved diagnosis, international commercialization, a new indication, or a transaction that changes distribution economics.

When does Cholbam lose exclusivity?

Cholbam’s seven-year U.S. orphan-drug exclusivity period ran from its 2015 approval and expired in March 2022. Orphan exclusivity prevents FDA approval of the same drug for the same indication during the exclusivity period, subject to statutory exceptions. It does not prevent all competing products, off-label use, compounding, or patents covering related formulations or methods.[4]

The end of orphan exclusivity did not automatically create a generic competitor. A generic applicant still must obtain FDA approval, demonstrate pharmaceutical equivalence and bioequivalence where applicable, address pediatric dosage-form requirements, and avoid or resolve any listed patent barriers.

Key exclusivity timeline

Event	Date or status
FDA approval	March 17, 2015
Seven-year orphan exclusivity begins	March 17, 2015
Orphan exclusivity expires	March 17, 2022
Patent protection	Potentially extends beyond orphan exclusivity
Pediatric granules	Creates formulation and administration complexity
Generic entry	No automatic entry on orphan-exclusivity expiration

What patents protect Cholbam?

Cholbam’s protection has historically relied on a combination of regulatory exclusivity, listed patents, formulation know-how, manufacturing controls, and specialized distribution.

The core intellectual-property questions concern:

• Cholic acid formulations suitable for pediatric administration.
• Methods of treating bile acid synthesis disorders.
• Dosing regimens and patient-selection criteria.
• Use in peroxisomal disorders.
• Product quality, particle size, granulation, and capsule or sachet manufacture.

The principal U.S. patent associated with Cholbam has been reported in FDA and patent records as covering methods of treating bile acid synthesis disorders with cholic acid. Patent term and listing status should be assessed against the current FDA Orange Book because expiry dates can reflect patent-term adjustment, pediatric extensions, terminal disclaimers, and delisting events.[5]

Patent-strength assessment

Protection layer	Strength	Commercial relevance
Orphan-drug exclusivity	Expired	Historically strong, no longer blocks approval
Core method-of-use patent	Moderate to strong until expiry	Can delay a labeled generic challenge
Cholic acid molecule	Weak as a composition claim	The molecule is naturally occurring and long known
Pediatric granule formulation	Potentially moderate	Raises development and manufacturing requirements
Manufacturing process	Moderate if undisclosed	Can increase practical entry cost
Distribution and reimbursement infrastructure	Moderate	Slows switching but does not create legal exclusivity

The estate is narrower than the patent estate of a new chemical entity. Its value comes from the rarity of the indication and the difficulty of recruiting enough patients for a commercial generic program, not from broad molecular patent coverage.

What generic entry risks exist for Cholbam?

Generic entry risk is real but likely gradual. The most probable first entrant would be a specialty generic manufacturer with experience in orphan products, pediatric formulations, or controlled distribution.

Paragraph IV challenge risk

A Paragraph IV applicant could challenge listed patents by asserting that the patents are invalid, unenforceable, or not infringed. The applicant would also need to address the scope of Cholbam’s approved indications and the evidentiary requirements for its dosage forms.

The commercial incentive for a Paragraph IV filing is limited by:

• A small number of eligible patients.
• High patient-acquisition costs.
• Need for metabolic-disease specialists.
• Product-specific pediatric administration requirements.
• Potential difficulty demonstrating bioequivalence for a formulation used in children.
• Risk that the reference product remains protected by a method-of-use patent.

A challenge could still be rational if the generic applicant expects premium pricing, obtains a first-filer advantage, or combines Cholbam with other orphan products in a portfolio.

Generic launch scenarios

Scenario	Probability profile	Market effect
No near-term generic	Base case	Brand maintains most treated patients
Authorized generic or private-label supply	Moderate commercial possibility	Price pressure without major clinical disruption
Single ANDA entrant	Moderate over the longer term	Discounting and payer-driven substitution
Multiple generic entrants	Low in the near term	Material price erosion and lower brand share
Patent settlement with delayed entry	Possible	Predictable erosion after agreed date

A generic would probably first affect new starts and price-sensitive payers. Existing patients with stable biochemical and clinical control may remain on Cholbam because physicians and families tend to avoid unnecessary changes in ultra-rare disease.

What is the Orange Book status of Cholbam?

Cholbam is an NDA product, making the FDA Orange Book the relevant source for listed patents and exclusivity information. The Orange Book does not capture every commercial barrier. It may not reflect trade secrets, manufacturing know-how, reimbursement controls, or all regulatory strategies used to defend a product.

The critical Orange Book diligence points are:

1. Current listed patent numbers.
2. Expiration dates after patent-term adjustment.
3. Whether listed patents cover all approved indications or only a subset.
4. Whether any patent has been withdrawn or delisted.
5. Whether a generic applicant has filed a Paragraph IV certification.
6. Whether litigation has triggered a 30-month stay under the Hatch-Waxman Act.

No broad public evidence indicates that Cholbam has faced the level of generic litigation seen with large primary-care products. The absence of high-profile litigation is consistent with the small market and limited economic incentive, but it does not remove future challenge risk.

Which companies are challenging Cholbam?

There has been no widely reported large-scale competitive challenge comparable to the patent contests surrounding major oncology, diabetes, or immunology products. The principal competitive threats are more likely to arise from:

• Specialty generic companies.
• Compounded cholic acid suppliers.
• Alternative bile-acid products used outside the labeled indication.
• Emerging therapies for the underlying genetic disorders.
• Supportive care that delays or reduces the need for treatment.

Compounding can create a practical price threat but does not necessarily provide an FDA-approved substitute with the same quality, labeling, and reimbursement profile. The legal and regulatory treatment of compounded cholic acid depends on the formulation, source, pharmacy status, and applicable FDA and state requirements.

How does Cholbam compare with Chenodal and competing bile-acid products?

Cholbam and Chenodal both contain cholic acid, but their commercial positioning differs by label and patient population.

Product	Active ingredient	Primary positioning	Market distinction
Cholbam	Cholic acid	Inherited bile acid synthesis disorders and peroxisomal disorders	Pediatric and ultra-rare metabolic focus
Chenodal	Chenodeoxycholic acid	Cerebrotendinous xanthomatosis	Separate orphan disease and treatment pathway
Ursodiol products	Ursodeoxycholic acid	Broader cholestatic and gallbladder-related uses	More established generic competition
Compounded bile acids	Variable	Patient-specific or off-label use	Regulatory and quality differences

Ursodiol is not a direct substitute for Cholbam in the approved genetic disorders. Chenodal also addresses a distinct biochemical defect. The limited interchangeability between products supports Cholbam’s pricing power, although payers may still impose utilization controls.

What licensing deals and ownership changes affect Cholbam?

Cholbam originated with Asklepion Pharmaceuticals. Retrophin acquired commercial rights and related assets, and Retrophin later changed its corporate name to Travere Therapeutics. These transactions placed Cholbam within a rare-disease portfolio that included other specialty and orphan products.[6]

The product’s strategic value in a transaction is based on:

• Recurring revenue from chronic patients.
• A specialist prescriber network.
• Regulatory history and safety data.
• Established specialty-pharmacy access.
• Manufacturing and quality systems.
• Cross-selling opportunities with other metabolic-disease products.

Cholbam is unlikely to drive a major pharmaceutical acquisition by itself. It is more suitable as an add-on asset in a portfolio transaction involving rare metabolic diseases, pediatric products, or specialty generics.

What patent litigation affects Cholbam?

No major public patent-litigation campaign has materially disrupted Cholbam’s market. The principal legal risk remains a future Hatch-Waxman challenge to any Orange Book-listed method-of-use or formulation patent.

Potential litigation issues include:

• Whether a proposed generic label induces infringement.
• Whether the patent claims cover the generic’s intended patient population.
• Whether a formulation patent is valid and properly listed.
• Whether a Paragraph IV notice triggers a 30-month stay.
• Whether the parties settle for a licensed or date-certain generic launch.

Because Cholbam’s market is small, settlement economics may favor a negotiated delayed-entry agreement over prolonged litigation. Any settlement would require scrutiny under FDA and Federal Trade Commission standards applicable to pharmaceutical patent settlements.

How strong is the Cholbam patent estate?

The patent estate is commercially useful but not exceptionally broad. The strongest defenses are likely to be indication-specific method claims, pediatric formulation claims, and practical barriers associated with producing a reliable dosage form for very young patients.

The weakest protection is the underlying cholic acid molecule itself. Since cholic acid is a known compound, exclusivity depends on the approved use, formulation, regulatory status, and execution of the commercial platform.

Overall assessment:

• Regulatory moat: historically strong, now reduced after orphan exclusivity expiration.
• Patent moat: moderate and time-limited.
• Manufacturing moat: moderate.
• Clinical switching moat: strong in stable patients.
• Commercial scale: limited.
• Generic attractiveness: moderate over time, low for mass-market manufacturers.

What is the likely financial outlook for Cholbam?

The base case is a mature orphan product with stable-to-declining revenue and high strategic value relative to absolute sales. Revenue can remain resilient if the owner maintains reimbursement support and captures newly diagnosed patients. Sustained growth would require one of four developments:

1. Greater genetic testing and earlier diagnosis.
2. Expanded reimbursement outside the United States.
3. Additional approved uses.
4. A distribution or licensing structure that lowers commercial cost per patient.

The downside case includes generic or compounded competition, payer restrictions, loss of specialist support, and patient attrition in severe peroxisomal disease. The upside case is limited by the size of the eligible population even if pricing remains strong.

Revenue exposure by risk factor

Risk factor	Expected effect
Generic approval	Lower price and new-start share
Loss of a core patent	Higher probability of ANDA entry
Payer tightening	Longer approval cycles and greater abandonment
Better diagnosis	Higher patient volume
Product transfer or licensing	Potentially lower operating cost
New disease-modifying therapy	Long-term substitution risk
Manufacturing disruption	High impact because supply options are limited

Key Takeaways

• Cholbam is an FDA-approved cholic acid product for ultra-rare inherited bile acid synthesis and peroxisomal disorders.
• Its orphan exclusivity expired in March 2022, but generic entry did not follow automatically.
• The product’s commercial defense rests on method-of-use and formulation protection, specialist prescribing, pediatric dosing requirements, and a small patient population.
• Cholbam has generated low-tens-of-millions annual revenue rather than blockbuster-scale sales.
• Generic risk is moderate over the long term but limited by small market size and formulation complexity.
• The product is strategically attractive as a recurring rare-disease asset but unlikely to be a standalone growth driver.
• The most important diligence items are current Orange Book listings, any Paragraph IV filing, ownership status, annual product sales, and payer access trends.

Frequently Asked Questions

Is Cholbam a biologic or a small-molecule drug?

Cholbam is a small-molecule oral drug containing cholic acid. Biosimilar risk does not apply. Any competitive approval would generally involve a generic or another cholic-acid product, not a biosimilar.

Can ursodiol replace Cholbam?

Ursodiol is not an FDA-approved interchangeable replacement for Cholbam’s labeled genetic disorders. Treatment decisions depend on the underlying enzyme defect, biochemical response, clinical status, and specialist oversight.

Does Cholbam have pediatric exclusivity?

The FDA label supports pediatric use, but pediatric exclusivity is a separate six-month extension that must be specifically granted by FDA action. It should not be assumed solely from pediatric labeling.

How is Cholbam reimbursed?

Coverage typically depends on commercial insurance, Medicaid, specialty-pharmacy authorization, and documentation of the qualifying disorder. Prior authorization can require genetic, biochemical, or specialist confirmation.

What would most increase Cholbam’s value?

The highest-value developments would be broader diagnosis, durable international reimbursement, an approved new indication, or a low-cost licensing structure that preserves pricing while reducing sales and distribution expense.

References

1. U.S. Food and Drug Administration. (2015). Cholbam (cholic acid) prescribing information. FDA.

2. Travere Therapeutics, Inc. (2023). Annual report pursuant to Section 13 or 15(d) of the Securities Exchange Act of 1934. U.S. Securities and Exchange Commission.

3. Travere Therapeutics, Inc. (2022). Annual report pursuant to Section 13 or 15(d) of the Securities Exchange Act of 1934. U.S. Securities and Exchange Commission.

4. U.S. Food and Drug Administration. (2024). Orphan drug designation and exclusivity. FDA.

5. U.S. Food and Drug Administration. (2024). Approved drug products with therapeutic equivalence evaluations: Orange Book. FDA.

6. Travere Therapeutics, Inc. (2024). Corporate and product portfolio disclosures. U.S. Securities and Exchange Commission.