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 are forty-four drug master file entries 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 Colorado, Denver	Phase 1/Phase 2
University of Nebraska	Phase 1/Phase 2
Children's Hospital Medical Center, Cincinnati	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/001250 Orphacol 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/002081 Cholic 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	Withdrawn	no	no	no	2015-11-20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 Analysis and Financial Projection Experimental

Market Dynamics and Financial Trajectory for CHOLBAM

Introduction

CHOLBAM, a bile acid medication, has been a significant player in the treatment of rare diseases, particularly bile acid synthesis disorders and peroxisomal disorders. Here, we delve into the market dynamics and financial trajectory of CHOLBAM, highlighting its key milestones, market performance, and future outlook.

FDA Approval and Initial Market Impact

CHOLBAM (cholic acid) was approved by the FDA in March 2015 for the treatment of bile acid synthesis disorders due to single enzyme defects and for the adjunctive treatment of patients with peroxisomal disorders[2][4][5].

Market Exclusivity

Following its approval, CHOLBAM was granted seven years of market exclusivity, which helped in establishing it as a leading treatment in its niche market[2].

Clinical Efficacy

The effectiveness of CHOLBAM has been demonstrated through clinical trials, showing improvements in laboratory parameters of liver function, weight gain, resolution of steatorrhea and hepatosplenomegaly, and prolonged survival in patients with bile acid synthesis disorders and peroxisomal disorders[5].

Acquisition and Portfolio Integration

In 2023, Mirum Pharmaceuticals acquired the rights and assets related to CHOLBAM from Travere Therapeutics, along with another bile acid medication, CHENODAL. This acquisition expanded Mirum’s portfolio and strengthened its position in the rare liver disease market[4].

Financial Performance

Revenue Growth

As part of Mirum Pharmaceuticals' portfolio, CHOLBAM contributed significantly to the company's financial growth. In 2023, Mirum reported total net product sales of $178.9 million, with CHOLBAM and CHENODAL contributing approximately $28 million in the fourth quarter alone[3].

Year-Over-Year Growth

The integration of CHOLBAM into Mirum’s portfolio was a key factor in the company’s 142% revenue growth over 2022. This growth underscores the market demand and the strategic value of CHOLBAM in Mirum’s rare disease treatment offerings[3].

Market Expansion

Global Reach

Mirum has expanded its international business to 18 countries, providing reimbursed access to CHOLBAM and other medications. This global reach has been crucial in increasing the drug's market penetration and revenue[1][3].

Label Expansion Opportunities

There are ongoing efforts to expand the indications for CHOLBAM and other Mirum medications. For instance, the company is anticipating label expansion opportunities for LIVMARLI and CHENODAL, which could indirectly benefit CHOLBAM by strengthening Mirum’s overall market position in rare liver diseases[1][3].

Regulatory and Pipeline Milestones

Upcoming Analyses and Submissions

In 2024, Mirum is expecting several key milestones, including the FDA PDUFA date for LIVMARLI in progressive familial intrahepatic cholestasis (PFIC) and the submission of a New Drug Application (NDA) for CHENODAL in cerebrotendinous xanthomatosis (CTX). While these are specific to other medications, they reflect the company's active pipeline and potential for further market expansion[1][3].

Financial Outlook

Revenue Projections

For 2024, Mirum expects continued revenue growth across all its commercial medicines, including CHOLBAM. The company projects global net product sales of $310 million to $320 million, indicating a strong financial trajectory[3].

Strategic Positioning

The acquisition of CHOLBAM has positioned Mirum as a leader in rare liver disease treatments. This strategic positioning, combined with a strong balance sheet and financial performance, sets the stage for sustained growth and market dominance[1][3].

Key Takeaways

• FDA Approval and Market Exclusivity: CHOLBAM was approved in 2015 with seven years of market exclusivity.
• Acquisition by Mirum: The drug was acquired by Mirum Pharmaceuticals in 2023, expanding its rare disease portfolio.
• Revenue Growth: CHOLBAM contributed to Mirum’s significant revenue growth in 2023.
• Global Expansion: The drug is now available in 18 countries with reimbursed access.
• Future Milestones: Mirum is anticipating several regulatory and pipeline milestones in 2024 that could further enhance CHOLBAM’s market position.

FAQs

Q: What is CHOLBAM used for? A: CHOLBAM is used for the treatment of bile acid synthesis disorders due to single enzyme defects and for the adjunctive treatment of patients with peroxisomal disorders.

Q: When was CHOLBAM approved by the FDA? A: CHOLBAM was approved by the FDA in March 2015.

Q: Who currently owns the rights to CHOLBAM? A: Mirum Pharmaceuticals acquired the rights to CHOLBAM from Travere Therapeutics in 2023.

Q: How has the acquisition of CHOLBAM impacted Mirum’s financial performance? A: The acquisition has contributed significantly to Mirum’s revenue growth, with total net product sales increasing by 142% over 2022.

Q: What are the future growth prospects for CHOLBAM? A: Mirum expects continued revenue growth across all its commercial medicines, including CHOLBAM, with projected global net product sales of $310 million to $320 million in 2024.

Sources

1. Mirum Pharmaceuticals Announces Preliminary Unaudited 2023 Net Revenue and Provides Corporate Updates - Biospace
2. U.S. Food and Drug Administration Approves Cholbam for the Treatment of Rare Diseases - Travere Therapeutics
3. Mirum Pharmaceuticals Reports Fourth Quarter and Year-End 2023 Financial Results and Provides Business Update - Biospace
4. Travere Therapeutics Completes Sale of Bile Acid Product Portfolio - Travere Therapeutics
5. FDA Approval Memo for CHOLBAM (cholic acid) capsules - FDA Access Data