MARALIXIBAT CHLORIDE - Generic Drug Details

What are the generic drug sources for maralixibat chloride and what is the scope of patent protection?

Maralixibat chloride is the generic ingredient in one branded drug marketed by Mirum and is included in one NDA. There are seven patents protecting this compound. Additional information is available in the individual branded drug profile pages.

Maralixibat chloride has one hundred and fourteen patent family members in twenty-two countries.

One supplier is listed for this compound.

Summary for MARALIXIBAT CHLORIDE

International Patents:	114
US Patents:	7
Tradenames:	1
Applicants:	1
NDAs:	1
Finished Product Suppliers / Packagers:	1
Raw Ingredient (Bulk) Api Vendors:	16
Clinical Trials:	2
Patent Applications:	47
What excipients (inactive ingredients) are in MARALIXIBAT CHLORIDE?	MARALIXIBAT CHLORIDE excipients list
DailyMed Link:	MARALIXIBAT CHLORIDE at DailyMed

DrugPatentWatch^® Estimated Loss of Exclusivity (LOE) Date for MARALIXIBAT CHLORIDE

Constraining patent/regulatory exclusivity:

TREATMENT OF CHOLESTATIC PRURITAS IN PATIENTS 12 MONTHS OF AGE TO LESS THAN 5 YEARS OF AGE WITH PROGRESSIVE FAMILIAL INTRAHEPATIC CHOLESTASIS (PFIC)

Dosage:

SOLUTION;ORAL

^*The generic entry opportunity date is the latter of the last compound-claiming patent and the last regulatory exclusivity protection. Many factors can influence early or later generic entry. This date is provided as a rough estimate of generic entry potential and should not be used as an independent source.

Recent Clinical Trials for MARALIXIBAT CHLORIDE

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

Sponsor	Phase
Takeda	Phase 3

See all MARALIXIBAT CHLORIDE clinical trials

Pharmacology for MARALIXIBAT CHLORIDE

Drug Class	Ileal Bile Acid Transporter Inhibitor
Mechanism of Action	Ileal Bile Acid Transporter Inhibitors Organic Anion Transporting Polypeptide 2B1 Inhibitors

Anatomical Therapeutic Chemical (ATC) Classes for MARALIXIBAT CHLORIDE

A05AX	Other drugs for bile therapy
A05A	BILE THERAPY
A05	BILE AND LIVER THERAPY
A	Alimentary tract and metabolism

US Patents and Regulatory Information for MARALIXIBAT CHLORIDE

Applicant	Tradename	Generic Name	Dosage	NDA	Approval Date	TE	Type	RLD	RS	Patent No.	Patent Expiration	Product	Substance	Delist Req.	Exclusivity Expiration
Mirum	LIVMARLI	maralixibat chloride	SOLUTION;ORAL	214662-001	Sep 29, 2021		RX	Yes	Yes
Mirum	LIVMARLI	maralixibat chloride	SOLUTION;ORAL	214662-002	Jul 24, 2024		RX	Yes	Yes	11,229,661
Mirum	LIVMARLI	maralixibat chloride	SOLUTION;ORAL	214662-001	Sep 29, 2021		RX	Yes	Yes
Mirum	LIVMARLI	maralixibat chloride	SOLUTION;ORAL	214662-001	Sep 29, 2021		RX	Yes	Yes	11,229,661

EU/EMA Drug Approvals for MARALIXIBAT CHLORIDE

Company	Drugname	Inn	Product Number / Indication	Status	Generic	Biosimilar	Orphan	Marketing Authorisation	Marketing Refusal
Mirum Pharmaceuticals International B.V.	Livmarli	Maralixibat chloride	EMEA/H/C/005857 Livmarli is indicated for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) 2 months of age and older.	Authorised	no	no	yes	2022-12-09

International Patents for MARALIXIBAT CHLORIDE

Country	Patent Number	Title	Estimated Expiration
Norway	2023023
World Intellectual Property Organization (WIPO)	2013063512
World Intellectual Property Organization (WIPO)	2020167981
Brazil	112021015815	Métodos para tratar colestase

Supplementary Protection Certificates for MARALIXIBAT CHLORIDE

Patent Number	Supplementary Protection Certificate	SPC Country	SPC Expiration	SPC Description
2771003	CR 2023 00017	Denmark		PRODUCT NAME: MARALIXIBAT ELLER ET FARMACEUTISK ACCEPTABELT SALT DERAF, ISAER MARALIXIBATCHLORID; REG. NO/DATE: EU/1/22/1704 20221212
2771003	301234	Netherlands		PRODUCT NAME: MARALIXIBAT OF EEN FARMACEUTISCH AANVAARDBAAR ZOUT DAARVAN, IN HET BIJZONDER MARALIXIBATCHLORIDE; REGISTRATION NO/DATE: EU/1/22/1704 20221212
2771003	CA 2023 00017	Denmark		PRODUCT NAME: MARALIXIBAT ELLER ET FARMACEUTISK ACCEPTABELT SALT DERAF, ISAER MARALIXIBATCHLORID; REG. NO/DATE: EU/1/22/1704 20221212
2771003	2023C/521	Belgium		PRODUCT NAME: MARALIXIBAT, OU UN SEL PHARMACEUTIQUEMENT ACCEPTABLE DE CELUI-CI, EN PARTICULIER LE CHLORURE DE MARALIXIBAT; AUTHORISATION NUMBER AND DATE: EU/1/22/1704 20221212

MARALIXIBAT CHLORIDE Market Analysis and Financial Projection Experimental

Market Dynamics and Financial Trajectory for Maralixibat Chloride (LIVMARLI)

Introduction

Maralixibat chloride, marketed as LIVMARLI, is a groundbreaking drug developed by Mirum Pharmaceuticals for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) and progressive familial intrahepatic cholestasis (PFIC). Here, we delve into the market dynamics and financial trajectory of this significant pharmaceutical product.

Regulatory Approvals and Expansion

LIVMARLI has garnered significant regulatory approvals that have propelled its market presence. It was approved by the U.S. Food and Drug Administration (FDA) in September 2021 for the treatment of cholestatic pruritus in patients with ALGS one year of age and older. Subsequently, it received approval for the same indication in several other countries, including Europe, where it is approved for patients two months and older[1][4].

In March 2024, LIVMARLI was approved by the FDA for the treatment of cholestatic pruritus in patients with PFIC five years of age and older, further expanding its market reach[2][4].

Commercial Performance

The commercial performance of LIVMARLI has been robust. In the first quarter of 2024, LIVMARLI net sales reached $42.8 million, representing a 47% growth compared to the first quarter of 2023. Global product sales for LIVMARLI grew 137% in the same period, contributing significantly to Mirum Pharmaceuticals' total revenue of $69.2 million for the quarter[2].

For the full year 2024, Mirum Pharmaceuticals is on track to achieve its revenue guidance of $310 to $320 million, driven largely by the continued demand for LIVMARLI[2].

International Expansion

Mirum Pharmaceuticals has been aggressively expanding the international market for LIVMARLI. The company has submitted a marketing authorization application (MAA) to the European Medicines Agency (EMA) for LIVMARLI for the treatment of cholestatic liver disease in patients with ALGS. Additionally, Mirum has entered into license and distribution agreements with several rare disease companies to commercialize LIVMARLI in countries such as South Korea, Greater China, and Israel[1].

Pipeline and Clinical Development

Beyond its current approvals, LIVMARLI is undergoing further clinical development. Mirum is conducting the MARCH-PFIC Phase 3 clinical trial to evaluate LIVMARLI in PFIC at a higher dose than in previous trials. The EMBARK Phase 2b clinical trial is also underway to evaluate LIVMARLI in biliary atresia (BA)[1].

For its second product candidate, volixibat, Mirum is conducting adaptive, potentially registrational, Phase 2b clinical trials for the treatment of primary sclerosing cholangitis (PSC), intrahepatic cholestasis of pregnancy (ICP), and primary biliary cholangitis (PBC)[1].

Financial Milestones and Agreements

Mirum Pharmaceuticals has made significant financial commitments to advance its product pipeline. The company has paid aggregate development and regulatory milestones of $51.0 million related to its LIVMARLI and volixibat programs. Additionally, Mirum is obligated to pay tiered sales milestone payments and royalties to Shire, ranging from low double-digits to mid-teens, based on certain net sales thresholds[1].

In 2023, Mirum entered into a definitive agreement with Travere Therapeutics to acquire all rights and assets related to two therapies, CHENODAL and CHOLBAM, for $210 million upfront and up to $235 million in potential tiered annual net sales-based milestones[5].

Cash Position and Operating Expenses

As of March 31, 2024, Mirum Pharmaceuticals had a cash balance of $302.8 million, indicating a strong financial position to support ongoing operations and future growth. Total operating expenses for the first quarter of 2024 were $95.7 million, which included $17.1 million of non-cash stock-based compensation and depreciation and amortization expense[2].

Market Growth Potential

The market for LIVMARLI is expected to grow significantly due to its unique position as the first and only treatment approved for cholestatic pruritus in patients with ALGS and PFIC. The annual growth rate for the target population is estimated at 2.73%, indicating a steady increase in potential patients[3].

Competitive Landscape

LIVMARLI's approval and market performance have established it as a leader in the treatment of rare liver diseases. Its unique mechanism as an ileal bile acid transporter (IBAT) inhibitor sets it apart from other treatments, providing a competitive edge in the market[4].

Key Takeaways

• Regulatory Approvals: LIVMARLI has received FDA approvals for ALGS and PFIC, with international approvals and submissions ongoing.
• Commercial Performance: Strong revenue growth driven by increasing demand for LIVMARLI.
• International Expansion: Agreements in place for commercialization in multiple countries.
• Pipeline Development: Ongoing clinical trials for additional indications.
• Financial Position: Strong cash balance and significant financial commitments to support growth.
• Market Growth: Steady growth potential due to unique market position and increasing patient population.

FAQs

Q: What is LIVMARLI used for? A: LIVMARLI (maralixibat chloride) is used for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) and progressive familial intrahepatic cholestasis (PFIC).

Q: When was LIVMARLI approved by the FDA? A: LIVMARLI was approved by the FDA in September 2021 for ALGS and in March 2024 for PFIC.

Q: What are the key financial milestones for Mirum Pharmaceuticals related to LIVMARLI? A: Mirum has paid $51.0 million in development and regulatory milestones and is obligated to pay tiered sales milestone payments and royalties to Shire.

Q: How is Mirum expanding the market for LIVMARLI internationally? A: Mirum has submitted marketing authorization applications and entered into license and distribution agreements with several rare disease companies to commercialize LIVMARLI in multiple countries.

Q: What other product candidates is Mirum developing? A: Mirum is developing volixibat for the treatment of primary sclerosing cholangitis (PSC), intrahepatic cholestasis of pregnancy (ICP), and primary biliary cholangitis (PBC).

Sources

1. Mirum Pharmaceuticals, Inc. - Annual Reports. Annual Reports.
2. Mirum Pharmaceuticals Reports First Quarter 2024 Financial Results and Provides Business Update. Biospace.
3. Summary of Key Model Parameters - Maralixibat (Livmarli). NCBI.
4. Takeda Submits New Drug Application for Maralixibat Chloride. Takeda.
5. Mirum Pharmaceuticals Reports Second Quarter 2023 Financial Results and Provides Business Update. Business Wire.