GIVLAARI Drug Patent Profile

Which patents cover Givlaari, and when can generic versions of Givlaari launch?

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

This drug has two hundred and seventy-nine patent family members in forty-five countries.

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

DrugPatentWatch^® Generic Entry Outlook for Givlaari

Givlaari was eligible for patent challenges on November 20, 2023.

By analyzing the patents and regulatory protections it appears that the earliest date for generic entry will be October 3, 2034. 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 GIVLAARI

International Patents:	279
US Patents:	9
Applicants:	1
NDAs:	1
Finished Product Suppliers / Packagers:	1
Clinical Trials:	1
Drug Prices:	Drug price information for GIVLAARI
Patent Litigation and PTAB cases:	See patent lawsuits and PTAB cases for GIVLAARI
What excipients (inactive ingredients) are in GIVLAARI?	GIVLAARI excipients list
DailyMed Link:	GIVLAARI at DailyMed

See drug prices for GIVLAARI

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

Constraining patent/regulatory exclusivity:

10,119,143

NDA:

212194

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 GIVLAARI

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

Sponsor	Phase
Alnylam Pharmaceuticals	Phase 3

See all GIVLAARI clinical trials

Pharmacology for GIVLAARI

Drug Class	Aminolevulinate Synthase 1-directed RNA Interaction Small Interfering RNA
Physiological Effect	Decreased RNA Integrity

US Patents and Regulatory Information for GIVLAARI

GIVLAARI is protected by nine US patents and one FDA Regulatory Exclusivity.

This potential generic entry date is based on patent 10,119,143.

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	GIVLAARI	givosiran sodium	SOLUTION;SUBCUTANEOUS	212194-001	Nov 20, 2019		RX	Yes	Yes	9,631,193
Alnylam Pharms Inc	GIVLAARI	givosiran sodium	SOLUTION;SUBCUTANEOUS	212194-001	Nov 20, 2019		RX	Yes	Yes	8,828,956		Y	Y
Alnylam Pharms Inc	GIVLAARI	givosiran sodium	SOLUTION;SUBCUTANEOUS	212194-001	Nov 20, 2019		RX	Yes	Yes	8,106,022		Y	Y
Alnylam Pharms Inc	GIVLAARI	givosiran sodium	SOLUTION;SUBCUTANEOUS	212194-001	Nov 20, 2019		RX	Yes	Yes	11,028,392		Y	Y
Alnylam Pharms Inc	GIVLAARI	givosiran sodium	SOLUTION;SUBCUTANEOUS	212194-001	Nov 20, 2019		RX	Yes	Yes	10,131,907		Y	Y
Alnylam Pharms Inc	GIVLAARI	givosiran sodium	SOLUTION;SUBCUTANEOUS	212194-001	Nov 20, 2019		RX	Yes	Yes	10,125,364		Y	Y
Alnylam Pharms Inc	GIVLAARI	givosiran sodium	SOLUTION;SUBCUTANEOUS	212194-001	Nov 20, 2019		RX	Yes	Yes	9,133,461		Y	Y

Expired US Patents for GIVLAARI

Applicant	Tradename	Generic Name	Dosage	NDA	Approval Date	Patent No.	Patent Expiration
Alnylam Pharms Inc	GIVLAARI	givosiran sodium	SOLUTION;SUBCUTANEOUS	212194-001	Nov 20, 2019	9,708,610
Alnylam Pharms Inc	GIVLAARI	givosiran sodium	SOLUTION;SUBCUTANEOUS	212194-001	Nov 20, 2019	10,273,477
Alnylam Pharms Inc	GIVLAARI	givosiran sodium	SOLUTION;SUBCUTANEOUS	212194-001	Nov 20, 2019	9,708,615
Alnylam Pharms Inc	GIVLAARI	givosiran sodium	SOLUTION;SUBCUTANEOUS	212194-001	Nov 20, 2019	9,150,605
Alnylam Pharms Inc	GIVLAARI	givosiran sodium	SOLUTION;SUBCUTANEOUS	212194-001	Nov 20, 2019	8,546,143
Alnylam Pharms Inc	GIVLAARI	givosiran sodium	SOLUTION;SUBCUTANEOUS	212194-001	Nov 20, 2019	11,530,408

International Patents for GIVLAARI

When does loss-of-exclusivity occur for GIVLAARI?

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

Argentina

Australia

Brazil

Canada

Chile

China

Costa Rica

Croatia

Cyprus

Denmark

Dominican Republic

Eurasian Patent Organization

European Patent Office

Guatemala

Hong Kong

Hungary

Israel

Japan

Lithuania

Malaysia

Mexico

Morocco

Netherlands

New Zealand

Norway

Peru

Philippines

Poland

Portugal

San Marino

Singapore

Slovenia

South Africa

South Korea

Spain

Taiwan

Tunisia

Ukraine

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 GIVLAARI around the world.

Country	Patent Number	Title	Estimated Expiration
World Intellectual Property Organization (WIPO)	03035868
Japan	2007314555	METHOD AND MEDICAMENT INHIBITING EXPRESSION OF SPECIFIC GENE
European Patent Office	1765074	PRODUITS DE SYNTHESE D'ARNsi MODIFIES EN POSITION (POSITIONALLY MODIFIED siRNA CONSTRUCTS)
Montenegro	02850
Argentina	090641
European Patent Office	3868883	COMPOSITIONS ET PROCÉDÉS D'INHIBITION D'EXPRESSION DU GÈNE ALAS1 (COMPOSITIONS AND METHODS FOR INHIBITING EXPRESSION OF THE ALAS1 GENE)
Hong Kong	1251014

Supplementary Protection Certificates for GIVLAARI

Patent Number	Supplementary Protection Certificate	SPC Country	SPC Expiration	SPC Description
3052628	PA2020527	Lithuania		PRODUCT NAME: GIVOSIRANAS ARBA JO FARMACINIU POZIURIU PRIIMTINA DRUSKA; REGISTRATION NO/DATE: EU/1/20/1428 20200302
3052628	C202030043	Spain		PRODUCT NAME: GIVOSIRAN O UNA SAL FARMACEUTICAMENTE ACEPTABLE DEL MISMO; NATIONAL AUTHORISATION NUMBER: EU/1/20/1428; DATE OF AUTHORISATION: 20200302; NUMBER OF FIRST AUTHORISATION IN EUROPEAN ECONOMIC AREA (EEA): EU/1/20/1428; DATE OF FIRST AUTHORISATION IN EEA: 20200302
3052628	301061	Netherlands		PRODUCT NAME: GIVOSIRAN OF EEN FARMACEUTISCH AANVAARDBAAR ZOUT DAARVAN; REGISTRATION NO/DATE: EU/1/20/1428 20200304
3052628	2020029	Norway		PRODUCT NAME: GIVOSIRAN ELLER ET FARMASOEYTISK AKSEPTABELT SALT DERAV; REG. NO/DATE: EU/1/20/1428 20200306
3052628	2090037-9	Sweden		PRODUCT NAME: GIVOSIRAN OR A PHARMACEUTICALLY ACCEPTABLE SALT THEREOF; REG. NO/DATE: EU/1/20/1428 20200304
3052628	20C1040	France		PRODUCT NAME: GIVOSIRAN OU L'UN DE SES SELS PHARMACEUTIQUEMENT ACCEPTABLES; NAT. REGISTRATION NO/DATE: EU/1/20/1428 20200304; FIRST REGISTRATION: - EU/1/20/1428 20200304
3052628	C03052628/01	Switzerland		PRODUCT NAME: GIVOSIRAN; REGISTRATION NO/DATE: SWISSMEDIC-ZULASSUNG 67895 29.03.2021

Market Dynamics and Financial Trajectory for GIVLAARI (Givosiran)

Last updated: July 27, 2025

Introduction

GIVLAARI (givosiran) is a groundbreaking therapeutic agent developed by Alnylam Pharmaceuticals for the treatment of acute hepatic porphyria (AHP), a rare, life-threatening genetic disorder characterized by recurrent, severe neurological attacks. Since its FDA approval in November 2019, GIVLAARI has positioned itself within a niche yet crucial segment of the rare disease pharmacopoeia. Its market performance and financial outlook are driven by a constellation of factors including clinical efficacy, regulatory environment, market demand, competitive landscape, and healthcare economics.

Market Overview: Rare Disease and AHP Landscape

The global rare disease market is expanding swiftly, driven by advancements in genomics, increased diagnosis rates, and the high unmet need for effective therapies. AHP affects approximately 5,000 to 10,000 individuals in the U.S. and Europe, with underdiagnosis common due to its complex symptomatology [1].

Current treatment options are limited; hemin infusions serve as supportive therapy but are expensive, invasive, and lack efficacy in preventing attacks. GIVLAARI offers a targeted RNA interference (RNAi) approach, reducing the hepatic accumulation of neurotoxic precursors, porphobilinogen (PBG) and delta-aminolevulinic acid (ALA). Its mechanism positions it as a first-in-class treatment, establishing a unique competitive advantage.

Market Dynamics

Regulatory and Payer Environment

Post-approval, GIVLAARI's reimbursement landscape has evolved, with payers increasingly recognizing its value based on clinical outcomes. In the U.S., payer policies have begun integrating GIVLAARI into formularies, albeit with notable prior authorization requirements and high cost-sharing, reflective of its premium pricing (~$575,000 annually per patient) [2].

Regulatory clarity in Europe remains progressing, with marketing authorization obtained in the European Union in 2020. However, reimbursement negotiations are ongoing, influencing market penetration.

Pricing and Access Challenges

The high cost of GIVLAARI underscores the ongoing debate over affordability and cost-effectiveness, especially considering the limited patient population. Health technology assessments (HTAs) in Europe and Medicaid/Medicare in the U.S. scrutinize its budget impact, potentially constraining access. Nonetheless, its ability to reduce attack frequency and hospitalizations provides a compelling value proposition for payers.

Patient Adoption and Market Penetration

Early uptake reflects cautious optimism; with approximately 1,000+ patients estimated to be treated globally by 2022 [3]. However, barriers such as diagnosis delays, administration logistics (subcutaneous injections), and clinician familiarity influence adoption rates. Expansion hinges on increasing awareness among specialists (e.g., hepatologists, geneticists).

Competitive Landscape

GIVLAARI's positioning as the first RNAi therapy for AHP grants it a leader’s edge. Nonetheless, competitors like Tebentafusp and emerging gene therapies targeting similar pathways pose long-term competitive threats. Additionally, the development of alternative modalities, such as monoclonal antibodies and gene editing, could influence future market shares.

Market Expansion Opportunities

Potential indications, such as prophylactic therapy for chronic porphyria symptoms and extended treatment to other hepatic metabolic disorders, remain under exploration. Moreover, applying RNAi technology to other rare conditions offers avenues for pipeline diversification.

Financial Trajectory

Revenue Trends and Key Drivers

Since its launch, GIVLAARI’s revenue growth has been steady but incremental. In 2021, Alnylam reported GIVLAARI sales exceeding $150 million globally, with U.S. sales representing approximately 80% of the total [4]. The catalog of patients treated in year one rapidly expanded as awareness increased.

Primary revenue drivers include:

• Patient volume growth: influenced by referrals from specialists.
• Pricing and reimbursement policies: adjustment of payer agreements influences net prices.
• Treatment adherence: consistent use supports revenue stability.

Cost Structure and Profitability Outlook

Development, manufacturing, and commercialization costs remain high given the complexity of the delivery technology. While Alnylam manages manufacturing economies, ongoing investments in clinician education, patient support programs, and regulatory filings influence margins.

Operationally, the company’s gross margins for GIVLAARI have hovered around 70%, with net margins constrained by R&D and commercialization expenses. Scalability and increased patient uptake are projected to improve profitability metrics over the coming years.

Forecasts and Growth Projections

Industry analyses project a compounded annual growth rate (CAGR) of approximately 15-20% over the next five years for the AHP-specific pharmaceutical market [5]. GIVLAARI is poised to benefit from this trend, with potential annual revenues reaching $300 million - $500 million by 2026 if market access expands and awareness improves.

Risks and Potential Disruptors

Despite promising growth, several risks threaten to temper projections:

• Pricing pressures: Heightened payer resistance could limit price increases.
• Regulatory hurdles: Delays or restrictions in certain markets.
• Emerging therapies: Future gene editing practices might supplant RNAi approaches.
• Market size constraints: The rare disease status caps patient volume, inherently limiting revenue ceiling.

Conclusion

GIVLAARI's rise within the niche AHP market reflects notable scientific innovation paired with strategic commercialization. Its financial trajectory is promising, supported by increasing patient treatment and favorable regulatory developments. However, sustained growth demands navigating payer dynamics, expanding awareness, and fostering continuous innovation. The long-term outlook suggests GIVLAARI will solidify its position as a first-in-class treatment, with significant growth potential aligned with the evolving landscape of personalized medicine for rare diseases.

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Key Takeaways

• GIVLAARI is the first RNAi therapy approved for AHP, serving a high unmet medical need.
• Its premium pricing creates both revenue opportunities and payer access challenges.
• Market expansion hinges on increasing diagnosis rates and overcoming reimbursement barriers.
• The global rare disease market is expanding, providing tailwinds for GIVLAARI’s growth.
• Long-term success depends on innovation, payer negotiations, and the evolving competitive landscape.

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FAQs

Q1: How does GIVLAARI compare to existing therapies for AHP?
A: GIVLAARI offers targeted, subcutaneous RNA interference therapy that reduces attack frequency, providing an alternative to traditional supportive treatments like hemin infusions, which are invasive and less effective in attack prevention.

Q2: What are the primary challenges in expanding GIVLAARI’s market?
A: Challenges include high treatment costs, payer reimbursement negotiations, limited patient awareness, and diagnosis delays inherent in rare diseases.

Q3: Are there any emerging competitors or alternative therapies for AHP?
A: Currently, GIVLAARI is first-in-class, but future gene therapies and other RNAi-based or monoclonal approaches are under development, posing potential competition.

Q4: What is the potential for expanding GIVLAARI’s indications?
A: Potential exists for prophylactic use in other hepatic metabolic disorders and exploring broader applications of RNA interference technology, although these are in early stages.

Q5: How critical is patient access to GIVLAARI’s commercial success?
A: Very critical; expanding access through improved reimbursement, awareness, and streamlined administration will drive sales growth and market penetration.

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References

1. National Organization for Rare Disorders. (2022). Acute hepatic porphyria (AHP).
2. Alnylam Pharmaceuticals. (2022). GIVLAARI Prescribing Information.
3. EvaluatePharma. (2022). Global Rare Disease Market Forecast.
4. Alnylam Annual Report. (2021).
5. Global Market Insights. (2022). RNAi Therapeutics Market Analysis.