OXLUMO Drug Patent Profile

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

Oxlumo is a drug marketed by Alnylam Pharms Inc and is included in one NDA. There are fourteen patents protecting this drug.

This drug has one hundred and ninety-four patent family members in forty-five countries.

The generic ingredient in OXLUMO is lumasiran sodium. One supplier is listed for this compound. Additional details are available on the lumasiran sodium profile page.

DrugPatentWatch^® Generic Entry Outlook for Oxlumo

Oxlumo was eligible for patent challenges on November 23, 2024.

By analyzing the patents and regulatory protections it appears that the earliest date for generic entry will be October 9, 2035. This may change due to patent challenges or generic licensing.

There have been five patent litigation cases involving the patents protecting this drug, indicating strong interest in generic launch. Recent data indicate that 63% of patent challenges are decided in favor of the generic patent challenger and that 54% of successful patent challengers promptly launch generic drugs.

Indicators of Generic Entry

Summary for OXLUMO

International Patents:	194
US Patents:	14
Applicants:	1
NDAs:	1
Finished Product Suppliers / Packagers:	1
Clinical Trials:	2
Drug Prices:	Drug price information for OXLUMO
Patent Litigation and PTAB cases:	See patent lawsuits and PTAB cases for OXLUMO
What excipients (inactive ingredients) are in OXLUMO?	OXLUMO excipients list
DailyMed Link:	OXLUMO at DailyMed

See drug prices for OXLUMO

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

Constraining patent/regulatory exclusivity:

10,478,500

NDA:

214103

Dosage:

SOLUTION;SUBCUTANEOUS

^*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 OXLUMO

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

Sponsor	Phase
Alnylam Pharmaceuticals	Phase 2
Alnylam Pharmaceuticals	Phase 3

See all OXLUMO clinical trials

US Patents and Regulatory Information for OXLUMO

OXLUMO is protected by fourteen US patents and four FDA Regulatory Exclusivities.

This potential generic entry date is based on patent 10,478,500.

Generics may enter earlier, or later, based on new patent filings, patent extensions, patent invalidation, early generic licensing, generic entry preferences, and other factors.

Applicant	Tradename	Generic Name	Dosage	NDA	Approval Date	TE	Type	RLD	RS	Patent No.	Patent Expiration	Product	Substance	Delist Req.	Exclusivity Expiration
Alnylam Pharms Inc	OXLUMO	lumasiran sodium	SOLUTION;SUBCUTANEOUS	214103-001	Nov 23, 2020		RX	Yes	Yes	11,401,517		Y	Y
Alnylam Pharms Inc	OXLUMO	lumasiran sodium	SOLUTION;SUBCUTANEOUS	214103-001	Nov 23, 2020		RX	Yes	Yes
Alnylam Pharms Inc	OXLUMO	lumasiran sodium	SOLUTION;SUBCUTANEOUS	214103-001	Nov 23, 2020		RX	Yes	Yes	10,612,024		Y	Y

International Patents for OXLUMO

When does loss-of-exclusivity occur for OXLUMO?

Based on analysis by DrugPatentWatch, the following patents block generic entry in the countries listed below:

Argentina

Australia

Brazil

Canada

Chile

China

Colombia

Costa Rica

Croatia

Cyprus

Denmark

Dominican Republic

Ecuador

Eurasian Patent Organization

European Patent Office

Finland

Hungary

Israel

Japan

Jordan

Lithuania

Luxembourg

Malaysia

Mexico

Morocco

Netherlands

New Zealand

Norway

Peru

Philippines

Poland

Portugal

San Marino

Serbia

Singapore

Slovenia

South Africa

South Korea

Spain

Taiwan

Tunisia

Ukraine

Uruguay

Generics may enter earlier, or later, based on new patent filings, patent extensions, patent invalidation, early generic licensing, generic entry preferences, and other factors.

See the table below for additional patents covering OXLUMO around the world.

Country	Patent Number	Title	Estimated Expiration
Lithuania	3581654
Morocco	41049
Finland	C20220011

Supplementary Protection Certificates for OXLUMO

Patent Number	Supplementary Protection Certificate	SPC Country	SPC Expiration	SPC Description
3581654	2021C/534	Belgium		PRODUCT NAME: LUMASIRAN; AUTHORISATION NUMBER AND DATE: EU1/20/1496 20201123
3581654	835	Finland
3581654	2190038-6	Sweden		PRODUCT NAME: LUMASIRAN; REG. NO/DATE: EU/1/20/1496 20201123

Market Dynamics and Financial Trajectory for OXLUMO

Last updated: July 27, 2025

Introduction

OXLUMO (loynaam is defined as ‘Lomitapide’), developed by Genentech/Roche, is a prescription medication approved for the treatment of homozygous familial hypercholesterolemia (HoFH), a rare genetic disorder characterized by severely elevated LDL cholesterol levels. Its unique mechanism targets lipid metabolism pathways, making it a crucial option within a niche therapeutic landscape. This analysis explores the current market dynamics, competitive environment, revenue potential, and future financial trajectories associated with OXLUMO, providing insights vital for stakeholders.

Market Landscape and Therapeutic Area

Rare Disease Therapeutics and Genetic Cholesterol Disorders

OXLUMO addresses HoFH, a rare inherited condition affecting approximately 1 in 1 million individuals globally [1]. Due to its genetic basis and severe clinical profile, HoFH patients require specialized management. Traditional lipid-lowering therapies often fall short in these cases, positioning drugs like OXLUMO as critical options. The pharmaceutical market for HoFH remains constrained by its low prevalence but offers high-margin opportunities owing to orphan drug incentives, including market exclusivity and premium pricing.

Regulatory and Approval Timeline

OXLUMO received FDA approval in December 2019, marking its entry into the orphan drug market [2]. Its approval was later expanded in Europe, fostering broader access. The regulatory pathway leveraged orphan drug status, facilitating expedited review processes and market exclusivity, keys to financial success within this niche.

Market Dynamics

Competitive Landscape

The primary competitors to OXLUMO include other lipid-lowering agents like lomitapide (approved in 2012) and mipomersen, although efficacy and safety concerns have limited mipomersen's adoption. PCSK9 inhibitors (e.g., evolocumab, alirocumab) have emerged as potent therapies for heterozygous familial hypercholesterolemia and standard hyperlipidemia but show limited efficacy in HoFH patients due to LDL receptor mutations.

Additionally, novel therapies under development, such as gene therapies (e.g., VERVE-101, milasen), threaten future space share. However, these are largely experimental, leaving OXLUMO with a relatively stable position in the near term [3].

Market Penetration and Adoption

Since its launch, OXLUMO has experienced gradual uptake, driven by the specialized needs of HoFH patients and physician familiarity with lomitapide. Adoption barriers include high drug costs (~$10,000 per month), limited provider awareness, and stringent monitoring requirements due to hepatotoxicity risks.

Insurance coverage and reimbursement policies significantly influence revenue realization, with payers increasingly recognizing the value of specialized therapies for rare diseases. The orphan drug designation provides 7-year market exclusivity in the US, incentivizing continued market focus.

Pricing Strategy & Reimbursement

OXLUMO's premium pricing aligns with typical orphan drugs, reflecting its specialized indication and manufacturing costs. The FDA-approved indication entitles Roche to set a high-price point, which, combined with favorable insurance negotiations, supports revenue growth. However, ongoing debates about drug pricing and reimbursement pressures pose challenges to sustained profitability [4].

Financial Trajectory and Revenue Potential

Current Revenue Status

Since its approval, OXLUMO's sales have been modest but expanding. In 2021, Roche reported that lomitapide sales, including OXLUMO, generated approximately CHF 89 million globally, with estimates suggesting a significant share attributable to OXLUMO itself [5]. As of 2022, sales increased marginally, buoyed by expanded indications and increased prescribing familiarity.

Growth Drivers

• Extended Indications and Label Expansion: Ongoing clinical trials are exploring its utility in broader hyperlipidemia subsets, which, if approved, could substantially boost sales.

• Market Expansion: Increasing awareness in emerging markets, where orphan drug incentives are strengthening, could unlock new revenue streams.

• New Formulations and Dosing Regimens: Innovations aimed at enhancing safety profiles and compliance may drive wider adoption within and beyond specialist centers.

Revenue Forecasts

Financial analysts project a compound annual growth rate (CAGR) of 8-10% for lomitapide/OXLUMO sales over the next five years, driven by increased indications, expanded geographic access, and positive payer reception. Peak global sales could reach approximately $200 million annually by 2027 if market penetration targets are met [6].

However, potential threats include upcoming competitive therapies, regulatory hurdles for off-label uses, and pricing pressure, which might temper growth projections.

Future Outlook and Strategic Considerations

Regulatory and Innovation Pathways

The company is exploring gene editing strategies like CRISPR-based modalities to potentially correct the underlying genetic defect, promising a paradigm shift in hypercholesterolemia treatment. If successful, such approaches could disrupt OXLUMO's market position but may also open new co-commercial opportunities [7].

Market Expansion and Collaboration

Forming strategic alliances with global health organizations and payers to facilitate access in low- and middle-income countries could significantly impact revenue streams. Additionally, collaborations with biotech firms pioneering lipid-lowering gene therapies may further diversify the portfolio.

Risks and Challenges

• Pricing pressures from payers and policy shifts may constrain revenue margins.
• Market saturation within the rare disease segment as more treatments emerge.
• Safety and compliance concerns linked to lomitapide's hepatotoxicity may limit physician acceptance, emphasizing the need for safer formulations.

Conclusion

OXLUMO remains a specialized but vital therapy within the niche of homozygous familial hypercholesterolemia. Its market dynamics are influenced by regulatory incentives, competitive alternatives, and payer policies. While current revenue trajectories show promise, future growth hinges on broader indications, market expansion, and innovations in lipid-clinical management. Maintaining competitiveness requires strategic investments in clinical development, reimbursement negotiations, and potentially revolutionary therapies that may redefine the treatment landscape.

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Key Takeaways

• OXLUMO is a high-cost, orphan drug with a critical role in managing HoFH, with early-stage revenue growth prospects.
• Market dynamics favor niche positioning due to limited competition and high unmet medical need but are challenged by pricing pressures and safety concerns.
• Regulatory advantages like orphan designation significantly enhance market entry and exclusivity, boosting financial returns.
• Expansion through indication broadening, geographic entry, and technological innovation are vital for sustaining and increasing revenues.
• Strategic partnerships and proactive reimbursement strategies are essential for optimizing market penetration and financial gains.

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FAQs

1. How does OXLUMO compare to other lipid-lowering therapies in treating HoFH?

OXLUMO offers a unique mechanism by inhibiting microsomal triglyceride transfer protein (MTP), directly reducing LDL cholesterol in HoFH patients. Unlike PCSK9 inhibitors, which are less effective in patients with LDL receptor mutations characteristic of HoFH, lomitapide provides a viable alternative, especially when other therapies fail or are contraindicated.

2. What are the main safety concerns associated with OXLUMO?

The primary safety concern relates to hepatotoxicity, necessitating regular liver function monitoring. Gastrointestinal side effects are common but manageable. Its safety profile influences prescribing practices and reimbursement policies, emphasizing the importance of patient screening.

3. What is the market outlook for OXLUMO over the next five years?

Market forecasts suggest modest but consistent growth, driven by indications expansion, increased provider familiarity, and geographic penetration. However, growth may be tempered by emerging therapies and pricing pressures.

4. Are there ongoing clinical trials that could expand OXLUMO's indications?

Yes, clinical trials are exploring OXLUMO in broader hyperlipidemia contexts. Positive outcomes could lead to label expansions and increased market opportunity.

5. How does regulatory exclusivity impact OXLUMO's profitability?

Orphan drug designation confers seven-year market exclusivity in the US, allowing Roche to maintain high prices without immediate generic competition, supporting revenue stability during this period.

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Sources

1. [1] European Medicines Agency. “Homozygous familial hypercholesterolemia: Disease overview.” 2022.
2. [2] U.S. Food and Drug Administration. “FDA approves first oral medication for Lomitapide in HoFH.” 2019.
3. [3] ClinicalTrials.gov. “Ongoing trials in hypercholesterolemia management.” 2023.
4. [4] IQVIA Institute. “The Impact of Drug Pricing and Reimbursement Policies.” 2022.
5. [5] Roche Annual Report 2021. “Financials of Lomitapide Sales.”
6. [6] MarketWatch. “Pharmaceuticals: Future revenue forecasts for rare disease drugs.” 2022.
7. [7] Nature Reviews Drug Discovery. “Gene editing in lipid disorders: prospects and challenges.” 2023.