OXLUMO Drug Patent Profile

When do Oxlumo patents expire, and what generic alternatives are available?

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-seven 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:	197
US Patents:	14
Applicants:	1
NDAs:	1
Patent Litigation and PTAB cases:	See patent lawsuits and PTAB cases for OXLUMO

US Patents and Regulatory Information for OXLUMO

OXLUMO is protected by fourteen US patents and two 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	10,612,024		Y	Y
Alnylam Pharms Inc	OXLUMO	lumasiran sodium	SOLUTION;SUBCUTANEOUS	214103-001	Nov 23, 2020		RX	Yes	Yes	8,106,022		Y	Y
Alnylam Pharms Inc	OXLUMO	lumasiran sodium	SOLUTION;SUBCUTANEOUS	214103-001	Nov 23, 2020		RX	Yes	Yes	11,261,447		Y	Y
Alnylam Pharms Inc	OXLUMO	lumasiran sodium	SOLUTION;SUBCUTANEOUS	214103-001	Nov 23, 2020		RX	Yes	Yes	10,612,027		Y	Y
Alnylam Pharms Inc	OXLUMO	lumasiran sodium	SOLUTION;SUBCUTANEOUS	214103-001	Nov 23, 2020		RX	Yes	Yes

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
Canada	3187050
Australia	2015330726
Israel	250448	חומרי רנ" א עם סליל כפול שעברו שינוי (Modified double-stranded rna agents)
South Africa	201702071
Japan	2019023241	標的化脂質 (TARGETED LIPID)
Japan	2023179412

Supplementary Protection Certificates for OXLUMO

Patent Number	Supplementary Protection Certificate	SPC Country	SPC Expiration	SPC Description
3204015	C202230012	Spain		PRODUCT NAME: LUMASIRAN, OPCIONALMENTE EN FORMA DE UNA SAL; NATIONAL AUTHORISATION NUMBER: EU/1/20/1496; DATE OF AUTHORISATION: 20201119; NUMBER OF FIRST AUTHORISATION IN EUROPEAN ECONOMIC AREA (EEA): EU/1/20/1496; DATE OF FIRST AUTHORISATION IN EEA: 20201119
3204015	2022C/509	Belgium		PRODUCT NAME: LUMASIRAN, INCLUSIEF ZOUTEN ERVAN; AUTHORISATION NUMBER AND DATE: EU/1/20/1496 20201123
3581654	C03581654/01	Switzerland		PRODUCT NAME: LUMASIRANUM; REGISTRATION NO/DATE: SWISSMEDIC-ZULASSUNG 68239 01.12.2021
3204015	C03204015/01	Switzerland		PRODUCT NAME: LUMASIRANUM; REGISTRATION NO/DATE: SWISSMEDIC-ZULASSUNG 68239 01.12.2021
3581654	202140021	Slovenia		PRODUCT NAME: LUMASIRAN; NATIONAL AUTHORISATION NUMBER: EU/1/20/1496/001; DATE OF NATIONAL AUTHORISATION: 20201119; AUTHORITY FOR NATIONAL AUTHORISATION: EU
3581654	122021000053	Germany		PRODUCT NAME: LUMASIRAN; REGISTRATION NO/DATE: EU/1/20/1496 20201119

OXLUMO (Lumasiran): Investment Scenario, Market Dynamics, and Financial Trajectory

Last updated: February 3, 2026

Summary

OXLUMO (lumasiran), a RNA interference (RNAi) therapeutic developed by Alnylam Pharmaceuticals, received FDA approval in November 2020 for the treatment of primary hyperoxaluria type 1 (PH1). As the first RNAi drug for this indication, OXLUMO's commercialization marks a significant milestone in precision medicine. This report analyzes the clinical and commercial prospects of OXLUMO, focusing on market dynamics, competitive landscape, revenue projections, regulatory developments, and investment considerations.

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What is the Pharmacological Profile of OXLUMO?

Parameter	Details
Active Ingredient	Lumasiran (RNA interference agent)
Mechanism of Action	Silences hepatic glycolate oxidase (GO) mRNA, reducing oxalate production in PH1
Indication	Primary hyperoxaluria type 1 (PH1)
Approval Date	November 2020 (FDA), December 2020 (EMA)
Administration Route	Subcutaneous injection
Dosing Regimen	Once monthly initially, then quarterly

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Market Landscape and Dynamics

Target Population and Epidemiology

Parameter	Data
Estimated Global PH1 Patients	~1,500 to 2,000 (per annum) (estimated)
U.S. Prevalence (adults + children)	Approx. 250-350 cases
Peak Onset Age	Childhood and early adolescence
Disease Burden	Chronic kidney disease, recurrent stones, oxalate nephropathy, high transplant risk

Market Drivers

• Unmet Medical Need: PH1 is a rare, life-threatening disease lacking targeted therapies until OXLUMO’s approval.
• Regulatory Milestones: Orphan drug designation and priority review facilitated earlier market entry.
• Potential Expansion: Ongoing studies suggest possibilities for broader indications, including other hyperoxalurias.

Market Challenges

• Pricing Policies: Orphan drugs often command high prices; OXLUMO’s initial pricing was ~$385,000 annually.
• Competitive Landscape: New therapies under development, such as CRISPR-based gene editing and other RNAi agents.
• Patient Access and Reimbursement: Insurance coverage variability could impact adoption rates.

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Commercial Performance and Revenue Projections

Initial Sales and Adoption Trends

| Year | Estimated Revenue | Growth Factors | Comments | |--------|----------------------+----------------------------------------------|----------------------------------------------------------| | 2020 | ~$5-10 million | Limited launch, initial uptake | Post-approval, rapid uptake in specialty centers | | 2021 | ~$30-50 million | Increased awareness, expanded clinics | Broader patient identification | | 2022+ | $100 million+ | Market expansion, pipeline integration | Potential for annual sales exceeding $500 million by 2025 |

Revenue Drivers

• Patient Access Growth: Expansion into Europe and other markets.
• Pricing and Reimbursement Optimization: Negotiations with payers.
• Pipeline Success: Long-term revenue sustainability hinges on pipeline progression.

Financial Outlook and Risks

Factors	Impact
Competitive Entering Markets	Downward pricing pressure
Regulatory Changes	Coverage policies, expedited approvals or delays
Pipeline Development	Success or failure of next-generation RNAi therapies

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Regulatory and Policy Environment

Policy Aspect	Impact
Orphan Drug Designation	Incentives, market exclusivity (7 years FDA)
Pricing Regulations	Possible U.S. and EU price controls
Reimbursement Policies	Variability across countries

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Competitive Landscape

Competitor/Agent	Development Stage	Mechanism	Market Position
Nedosiran (DCR-PHXC, Dicerna)	FDA-approved (2021)	RNAi, targets hydrophilic enzyme	Competing RNAi for PH1
CRISPR-based therapies	Preclinical/early	Gene editing	Future potential disruptor
Small molecule inhibitors	Early-stage	Undetermined	Unlikely given current genetics focus

Note: OXLUMO has a first-mover advantage given its approval status and specific targeting of PH1.

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Future Growth Opportunities

Opportunity	Description
Broadened Patient Access	Expansion into non-U.S. markets, especially Europe and Japan
Indications Expansion	Potential for other hyperoxaluria types or metabolic disorders
Pipeline Development	Next-generation RNAi agents, gene editing, combination therapies

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Valuation and Investment Considerations

Aspect	Status
Market Penetration Expectations	Moderate to high growth expected in the next 3–5 years
Pricing Strategy	Premium pricing sustained by orphan designation and chronic need
Cost Dynamics	High R&D and operational costs balanced by revenue potential
Patent and Exclusivity	Patent life until at least 2030, ensuring market protection

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Comparison with Similar Therapies

Attribute	OXLUMO	Nedosiran (Dicerna)	Gene Editing (CRISPR) prototypes
Approval Status	Approved (FDA/EMA)	Pending approval	Preclinical/early trials
Indication	PH1	PH1	PH1 and broader hyperoxalurias
Pricing	~$385,000/year	Estimated similar	Not priced yet
Market Position	First Approved RNAi	Competitor, second-mover	Future disruptor

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Regulatory Outlook and Pipeline Progress

Timeline	Milestone	Relevance
2023-2024	Additional approvals in Europe, Japan	Market expansion
2024+	Evidence for broader indications	Revenue growth
2025+	Potential pipeline approvals	Long-term dominance

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Key Challenges and Risks

Risk Factor	Impact
Market Penetration Pace	Slower adoption may limit revenues
Competitive Innovation	New therapies could threaten market share
Reimbursement Hurdles	Impact on patient access and revenue
Regulatory Changes	Potential requirement for new approvals or pricing controls

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Key Takeaways

• First-mover advantage positions OXLUMO strongly within the orphan drug space for PH1.
• Revenue projections suggest significant growth potential, with forecasted revenues exceeding $500 million annually by 2025.
• Market expansion is pivotal, especially into Europe and other jurisdictions, contingent on regulatory approvals.
• Pricing strategy sustains high margins and reflects the unmet need for targeted therapies in rare diseases.
• Pipeline development and competition could influence OXLUMO’s long-term market dominance.

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FAQs

Q1: What is the primary competitive advantage of OXLUMO?
OXLUMO is the first FDA-approved RNAi therapeutic targeting primary hyperoxaluria type 1 (PH1), providing a molecularly targeted approach that directly reduces oxalate precursors, offering significant clinical benefits over previous non-specific treatments.

Q2: How does OXLUMO's market potential compare with other orphan drugs?
OXLUMO's peak sales forecast exceeds $500 million annually by 2025, comparable to successful orphan therapies like Spinraza (€1.3 billion peak in neuromuscular indications), driven by high unmet need, specialized patient populations, and exclusive marketing rights.

Q3: What regulatory developments could impact OXLUMO's commercialization?
Further approvals in international markets, potential label expansions, or changes in pricing and reimbursement policies may influence revenue trajectories.

Q4: Are there significant pipeline threats to OXLUMO?
Yes, gene editing therapies and next-generation RNAi agents under development could challenge OXLUMO's market position if proven safe, effective, and commercially viable.

Q5: What strategic moves should investors monitor regarding OXLUMO?
Attention to pipeline advancements, regulatory approvals outside the U.S., reimbursement negotiations, and competitive launches will inform future investment decisions.

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References

[1] FDA. (2020). FDA approves first treatment for primary hyperoxaluria type 1.
[2] Alnylam Pharmaceuticals. (2022). OXLUMO (lumasiran) prescribing information.
[3] European Medicines Agency. (2020). OXLUMO approval letter.
[4] MarketWatch. (2021). OXLUMO revenue estimates.
[5] Global Data. (2022). Rare disease market analysis.