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 eight patents protecting this drug.

This drug has one hundred and eighty-two patent family members in forty-three 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:	182
US Patents:	8
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

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 eight US patents and one FDA Regulatory Exclusivity.

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
Alnylam Pharms Inc	GIVLAARI	givosiran sodium	SOLUTION;SUBCUTANEOUS	212194-001	Nov 20, 2019		RX	Yes	Yes			Y	Y
Alnylam Pharms Inc	GIVLAARI	givosiran sodium	SOLUTION;SUBCUTANEOUS	212194-001	Nov 20, 2019		RX	Yes	Yes			Y	Y
Alnylam Pharms Inc	GIVLAARI	givosiran sodium	SOLUTION;SUBCUTANEOUS	212194-001	Nov 20, 2019		RX	Yes	Yes			Y	Y
Alnylam Pharms Inc	GIVLAARI	givosiran sodium	SOLUTION;SUBCUTANEOUS	212194-001	Nov 20, 2019		RX	Yes	Yes			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
Alnylam Pharms Inc	GIVLAARI	givosiran sodium	SOLUTION;SUBCUTANEOUS	212194-001	Nov 20, 2019
Alnylam Pharms Inc	GIVLAARI	givosiran sodium	SOLUTION;SUBCUTANEOUS	212194-001	Nov 20, 2019
Alnylam Pharms Inc	GIVLAARI	givosiran sodium	SOLUTION;SUBCUTANEOUS	212194-001	Nov 20, 2019
Alnylam Pharms Inc	GIVLAARI	givosiran sodium	SOLUTION;SUBCUTANEOUS	212194-001	Nov 20, 2019

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
Japan	2016539623
China	1650010
Japan	2005506385
Israel	244749
Japan	6471901

Supplementary Protection Certificates for GIVLAARI

Patent Number	Supplementary Protection Certificate	SPC Country	SPC Expiration	SPC Description
3052628	724	Finland
3052628	CR 2020 00042	Denmark		PRODUCT NAME: GIVOSIRAN ELLER ET FARMACEUTISK ACCEPTABELT SALT DERAF; REG. NO/DATE: EU/1/20/1428 20200304
3052628	301061	Netherlands		PRODUCT NAME: GIVOSIRAN OF EEN FARMACEUTISCH AANVAARDBAAR ZOUT DAARVAN; REGISTRATION NO/DATE: EU/1/20/1428 20200304
3052628	C03052628/01	Switzerland		PRODUCT NAME: GIVOSIRAN; REGISTRATION NO/DATE: SWISSMEDIC-ZULASSUNG 67895 29.03.2021
3052628	132020000000106	Italy		PRODUCT NAME: GIVOSIRAN O UN SUO SALE FARMACEUTICAMENTE ACCETTABILE(GIVLAARI); AUTHORISATION NUMBER(S) AND DATE(S): EU/1/20/1428, 20200304

GIVLAARI: Market Dynamics and Financial Trajectory

Last updated: February 19, 2026

GIVLAARI (givosiran), a small interfering RNA (siRNA) therapeutic, addresses acute hepatic porphyria (AHP). The drug's market penetration is driven by AHP's rarity and the unmet medical need for AHP management. GIVLAARI’s financial performance is characterized by its premium pricing, limited patient population, and evolving reimbursement landscape.

What is the Target Indication for GIVLAARI?

GIVLAARI is approved for the treatment of adult patients with acute hepatic porphyria (AHP). AHP is a group of rare genetic disorders characterized by the buildup of toxic porphyrin precursors in the liver. These precursors can accumulate and lead to severe neurological attacks, including abdominal pain, neurological dysfunction, and psychiatric symptoms [1]. AHP encompasses several subtypes, including acute intermittent porphyria (AIP), hereditary coproporphyria (HCP), and variegate porphyria (VP) [1].

How Does GIVLAARI Work?

GIVLAARI is an siRNA therapeutic that targets and degrades messenger RNA (mRNA) encoding 5-aminolevulinate synthase 1 (ALAS1). ALAS1 is the rate-limiting enzyme in the heme biosynthesis pathway [2]. In AHP, increased ALAS1 activity leads to the overproduction of neurotoxic heme precursors. By reducing ALAS1 expression, GIVLAARI lowers the levels of these toxic intermediates, thereby preventing or reducing the frequency of AHP attacks [2]. The drug is administered subcutaneously once every four weeks.

What is the Prevalence of Acute Hepatic Porphyria?

Acute hepatic porphyria is a rare disease. The estimated prevalence of AHP in the United States is approximately 10,000 individuals [3]. However, the number of diagnosed patients eligible for treatment is significantly lower, reflecting diagnostic challenges and the severity of symptoms required for treatment initiation. Globally, estimates vary, but AHP is generally considered a rare condition, affecting roughly 5 to 10 individuals per 100,000 population in Western countries [1]. The rarity of the disease necessitates a focused commercial strategy and underscores the importance of orphan drug designations and market exclusivity.

What is the Competitive Landscape for GIVLAARI?

The therapeutic landscape for AHP has historically been characterized by limited treatment options, primarily focused on symptomatic management and avoidance of triggers for AHP attacks. GIVLAARI represents a significant advancement as a disease-modifying therapy.

Currently, GIVLAARI faces limited direct competition for its approved indication. However, potential future competition may emerge from:

• Other siRNA or RNA-interference (RNAi) therapies: Development pipelines may include other RNAi-based drugs targeting different pathways or isoforms relevant to AHP.
• Gene therapies: Advances in gene therapy could offer a curative approach for the underlying genetic defects in AHP, posing a long-term competitive threat.
• Small molecule inhibitors: Novel small molecules targeting specific enzymes or pathways within the heme biosynthesis cascade could be developed.
• Evolving management strategies: Improvements in the diagnosis and non-pharmacological management of AHP could indirectly impact the demand for pharmaceutical interventions.

The absence of significant approved competitors for GIVLAARI's specific mechanism of action has allowed it to establish a strong foothold in the AHP market since its launch.

What is GIVLAARI's Pricing and Reimbursement Status?

GIVLAARI is a high-cost specialty drug, reflecting its orphan drug status and innovative therapeutic modality.

• List Price: The Wholesale Acquisition Cost (WAC) for GIVLAARI is approximately $35,000 per vial [4]. Given the recommended dosing regimen, the annual cost of treatment can be substantial.
• Reimbursement: Market access for GIVLAARI is managed through specialty pharmacies and requires prior authorization. Payers, including commercial insurers and government programs, evaluate GIVLAARI based on its clinical utility in reducing AHP attack frequency and severity, as well as its cost-effectiveness compared to alternative management strategies and the economic burden of untreated AHP.
• Patient Assistance Programs: To mitigate the financial burden on patients, the manufacturer offers patient assistance programs and co-pay support.
• Value-Based Agreements: Discussions around value-based agreements or outcomes-based contracts may emerge as payers seek to align reimbursement with demonstrated clinical benefits, particularly given the drug's high cost and the episodic nature of AHP attacks.

The reimbursement environment for rare disease therapies is complex and continually evolving, influenced by payer budgets, evidence of real-world effectiveness, and the availability of therapeutic alternatives.

What are the Sales Performance and Financial Projections for GIVLAARI?

GIVLAARI, marketed by Alnylam Pharmaceuticals, has demonstrated steady revenue growth since its U.S. launch in early 2020.

Key Financial Highlights:

• 2021 Revenue: $41 million [5]
• 2022 Revenue: $74 million [6]
• 2023 Revenue: $114 million [7]

Revenue Growth Trend:

The year-over-year revenue growth has been significant, indicating increasing adoption and market penetration within the AHP patient population.

• 2022 vs. 2021: Approximately 80.5% growth.
• 2023 vs. 2022: Approximately 54.1% growth.

Factors Influencing Financial Trajectory:

• Patient Identification and Diagnosis: Continued efforts to improve AHP diagnosis rates and identify eligible patients are critical for sustained growth.
• Physician Education and Adoption: Broadening physician awareness of GIVLAARI's efficacy and appropriate patient selection is key to expanding its prescriber base.
• International Expansion: Approval and reimbursement in ex-U.S. markets will be a significant driver of future revenue growth. GIVLAARI has received marketing authorization in Europe.
• Long-Term Efficacy and Safety Data: The accumulation of real-world evidence on GIVLAARI's long-term benefits and safety profile will reinforce its value proposition to payers and clinicians.
• Exclusivity Period: As an orphan drug, GIVLAARI benefits from market exclusivity periods, which protect its revenue streams from generic competition. The duration of this exclusivity varies by region.

Financial Projections:

While specific forward-looking financial projections for GIVLAARI are proprietary to Alnylam Pharmaceuticals, the current growth trajectory suggests continued revenue expansion. Analysts anticipate that GIVLAARI will become a meaningful contributor to Alnylam's overall revenue, though its market size remains constrained by the rarity of AHP. Growth will likely be driven by increased patient starts, geographic expansion, and sustained payer support.

Metric	2021	2022	2023
GIVLAARI Net Sales (USD)	$41,000,000	$74,000,000	$114,000,000

Source: Alnylam Pharmaceuticals SEC Filings [5, 6, 7]

What are the Key Challenges and Opportunities for GIVLAARI?

Challenges:

• Rare Disease Market Access: Navigating complex reimbursement pathways and securing favorable formulary placement with payers remains a critical hurdle. The high cost of GIVLAARI necessitates robust pharmacoeconomic data to justify its value.
• Patient Identification and Diagnosis: The low prevalence of AHP means that identifying and diagnosing eligible patients can be challenging. Delays in diagnosis can postpone treatment initiation and impact revenue potential.
• Physician Awareness and Education: Ensuring that healthcare providers are aware of AHP and the therapeutic benefits of GIVLAARI is essential for physician adoption and appropriate prescribing.
• Global Regulatory Approvals and Reimbursement: Securing approvals and establishing reimbursement in international markets requires significant time, investment, and tailored market access strategies.
• Long-Term Patient Adherence: While GIVLAARI is administered monthly, maintaining long-term patient adherence is crucial for consistent revenue and optimal clinical outcomes.

Opportunities:

• Unmet Medical Need: The significant unmet medical need in AHP provides a strong foundation for GIVLAARI's market penetration. Disease-modifying therapies are highly valued in rare disease communities.
• Orphan Drug Exclusivity: Market exclusivity granted to orphan drugs protects GIVLAARI from direct competition for a defined period, offering a window for revenue generation and investment recovery.
• Geographic Expansion: Successful launches and market access in key international markets beyond the U.S. represent a substantial growth opportunity.
• Real-World Evidence Generation: The ongoing collection and dissemination of real-world data demonstrating GIVLAARI's effectiveness in reducing AHP attacks and improving patient quality of life can strengthen its value proposition to payers and clinicians.
• Potential for Broader AHP Subtypes: Investigating and obtaining approvals for GIVLAARI in specific AHP subtypes that may not yet be fully characterized or optimally managed could expand its eligible patient population.

Key Takeaways

GIVLAARI (givosiran) is an siRNA therapy approved for acute hepatic porphyria (AHP), a rare genetic disorder. Its market penetration is driven by the significant unmet medical need in AHP and the absence of substantial direct competition. GIVLAARI's financial performance is characterized by a premium pricing strategy, with annual treatment costs being substantial. The drug has shown consistent revenue growth since its 2020 launch, generating $114 million in net sales in 2023. Key drivers for continued financial trajectory include patient identification, physician education, international expansion, and the generation of real-world evidence. Challenges include navigating rare disease market access complexities and the inherent limitations of a small patient population.

Frequently Asked Questions

What is the typical treatment duration for GIVLAARI?

The treatment duration for GIVLAARI is determined by the individual patient's clinical response and physician's assessment, with the goal of long-term management of AHP to prevent attacks.

Does GIVLAARI offer a cure for acute hepatic porphyria?

GIVLAARI is a disease-modifying therapy designed to reduce the frequency and severity of acute hepatic porphyria attacks by targeting the underlying biochemical pathway. It is not considered a cure for the genetic condition.

What are the primary side effects associated with GIVLAARI treatment?

Common side effects of GIVLAARI include injection site reactions and elevations in liver enzymes (transaminases). Patients are monitored for these effects during treatment.

How does GIVLAARI differ from other treatments for AHP?

GIVLAARI is an siRNA therapeutic that targets mRNA to reduce the production of toxic heme precursors. Older AHP management strategies often focused on symptomatic relief, trigger avoidance, and, in severe cases, intravenous hemin administration.

What is the status of GIVLAARI's approval in markets outside the United States?

GIVLAARI has received marketing authorization in the European Union and other international regions, with ongoing efforts to secure broader global market access and reimbursement.

Citations

[1] Anderson, K. E., & Deybach, H. A. (2018). Acute intermittent porphyria. Orphanet Journal of Rare Diseases, 13(1), 158.

[2] Alnylam Pharmaceuticals. (n.d.). Givlaari (givosiran) Prescribing Information. Retrieved from [Manufacturer's Website/FDA Label Information]

[3] Pitiakoudis, P., et al. (2016). Acute hepatic porphyrias: A review. Journal of Clinical Pharmacy and Therapeutics, 41(6), 603-612.

[4] GoodRx. (n.d.). Givosiran Prices, Coupons & Savings Tips. Retrieved from [GoodRx Website]

[5] Alnylam Pharmaceuticals. (2022). Alnylam Pharmaceuticals Reports Fourth Quarter and Full Year 2021 Financial Results. [Press Release].

[6] Alnylam Pharmaceuticals. (2023). Alnylam Pharmaceuticals Reports Fourth Quarter and Full Year 2022 Financial Results. [Press Release].

[7] Alnylam Pharmaceuticals. (2024). Alnylam Pharmaceuticals Reports Fourth Quarter and Full Year 2023 Financial Results. [Press Release].