GIVOSIRAN SODIUM - Generic Drug Details

What are the generic drug sources for givosiran sodium and what is the scope of freedom to operate?

Givosiran sodium is the generic ingredient in one branded drug marketed by Alnylam Pharms Inc and is included in one NDA. There are nine patents protecting this compound. Additional information is available in the individual branded drug profile pages.

Givosiran sodium has two hundred and seventy-nine patent family members in forty-five countries.

One supplier is listed for this compound.

Summary for GIVOSIRAN SODIUM

International Patents:	279
US Patents:	9
Tradenames:	1
Applicants:	1
NDAs:	1
Finished Product Suppliers / Packagers:	1
Patent Litigation and PTAB cases:	See patent lawsuits and PTAB cases for GIVOSIRAN SODIUM
What excipients (inactive ingredients) are in GIVOSIRAN SODIUM?	GIVOSIRAN SODIUM excipients list
DailyMed Link:	GIVOSIRAN SODIUM at DailyMed

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

Constraining patent/regulatory exclusivity:

10,119,143

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.

Pharmacology for GIVOSIRAN SODIUM

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

Anatomical Therapeutic Chemical (ATC) Classes for GIVOSIRAN SODIUM

A16AX	Various alimentary tract and metabolism products
A16A	OTHER ALIMENTARY TRACT AND METABOLISM PRODUCTS
A16	OTHER ALIMENTARY TRACT AND METABOLISM PRODUCTS
A	Alimentary tract and metabolism

US Patents and Regulatory Information for GIVOSIRAN SODIUM

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	11,028,392		Y	Y
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
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	10,125,364		Y	Y

Expired US Patents for GIVOSIRAN SODIUM

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

International Patents for GIVOSIRAN SODIUM

Country	Patent Number	Title	Estimated Expiration
Canada	3169218	COMPOSITIONS ET PROCEDES PERMETTANT D'INHIBER L'EXPRESSION DU GENE ALAS1 (COMPOSITIONS AND METHODS FOR INHIBITING EXPRESSION OF THE ALAS1 GENE)
World Intellectual Property Organization (WIPO)	2005121370
Australia	2003295387	MODIFIED OLIGONUCLEOTIDES FOR USE IN RNA INTERFERENCE
Mexico	2022001017
Morocco	39000

Supplementary Protection Certificates for GIVOSIRAN SODIUM

Patent Number	Supplementary Protection Certificate	SPC Country	SPC Expiration	SPC Description
3052628	C03052628/01	Switzerland		PRODUCT NAME: GIVOSIRAN; REGISTRATION NO/DATE: SWISSMEDIC-ZULASSUNG 67895 29.03.2021
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	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

Market Dynamics and Financial Trajectory for Givosiran Sodium

Last updated: July 27, 2025

Introduction

Givosiran sodium (commercially known as Givlaari) represents a significant development within the landscape of rare disease therapeutics, specifically targeting acute hepatic porphyria (AHP). As a first-in-class small interfering RNA (siRNA) therapeutic, its market trajectory is shaped by factors including unmet clinical needs, regulatory pathways, competitive landscape, pricing strategies, and evolving healthcare policies. This comprehensive analysis explores the current market dynamics and forecasts the financial trajectory of givosiran sodium, providing critical insights for stakeholders.

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Therapeutic Profile and Clinical Positioning

Givosiran sodium is designed to inhibit hepatic aminolevulinic acid synthase 1 (ALAS1), thereby reducing the accumulation of toxic porphyrin precursors responsible for AHP attacks. Approved by the U.S. FDA in 2019, it addresses a rare, debilitating genetic disorder characterized by episodes of severe neurological and visceral symptoms[^1].

Its clinical benefit hinges on a targeted mechanism, reducing attack frequency and improving quality of life. The orphan drug designation supports exclusive marketing rights, incentivizing development and commercialization efforts.

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Market Size and Unmet Needs

AHP affects approximately 5,000–8,000 individuals in the U.S. and Europe[^2], with many patients underdiagnosed. The disease’s episodic nature results in significant morbidity, healthcare utilization, and economic burden. The limited standard of care, primarily supportive management, underscores the unmet need for disease-modifying therapy.

Consequently, the initial market opportunity, while niche, possesses high value given the severity of the disease and lack of alternatives. The low prevalence complicates large-scale sales but enhances premium pricing potential under orphan drug conventions.

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Market Dynamics and Drivers

1. Regulatory Approvals and Reimbursement Policies

Post-approval, givosiran has benefited from rapid regulatory pathways, with orphan drug designation expediting approval. Reimbursement, however, remains contingent on cost-effectiveness assessments in various jurisdictions. Payers are increasingly scrutinizing high-cost therapies, influencing net revenue realization.

2. Adoption and Prescribing Trends

Clinician awareness remains crucial. Early adopters and specialists in metabolic and hematological disorders are primary prescribers, with ongoing educational initiatives expanding access. Adoption rates depend on demonstrated real-world efficacy, safety profile, and patient adherence.

3. Competitive Landscape

Currently, givosiran faces minimal direct competition; however, emerging therapies and gene editing approaches could challenge its market share. For example, lyophilized gene therapy candidates and small molecule alternatives are in preclinical or early clinical stages[^3].

4. Pricing Strategy and Market Penetration

Givosiran's pricing, approximately $575,000 annually per patient in the U.S., reflects its orphan designation and clinical value. Pricing negotiations, discounts, and patient assistance programs influence net revenue. Market penetration accelerates as the therapy proves durable efficacy and safety in broader populations.

5. Manufacturing and Supply Chain Considerations

Manufacturing complexities associated with siRNA therapeutics impact costs and scaling. Ensuring reliable supply chains is vital for sustained growth and meeting demand.

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Financial Trajectory: Forecast and Outlook

Revenue Projections

Based on current prescriber uptake, historical sales data, and market size estimates, revenues are expected to follow an initial rapid growth phase, subsequently plateauing as market saturation approaches (see Figure 1).

Figure 1: Givosiran Revenue Trajectory (Estimated)

Year	Estimated Revenue (USD millions)	Key Assumptions
2023	250	Steady prescriber adoption, ongoing reimbursement contracts
2024	370	Expanded geographic access, increased awareness
2025	500	Market saturation, stable prescriber base
2026	530	Competitive pressures, potential late-stage pipeline entrants

This trajectory accommodates forecasted growth, tempered by constraints including reimbursement limits, patient eligibility, and competitive innovations.

Profitability and Cost Considerations

High R&D expenses anticipated from ongoing pipeline development, coupled with manufacturing costs typical of biologics and siRNA drugs, influence gross margins. Cost containment and economies of scale are projected to improve profitability over time.

Market Expansion and Future Opportunities

Registration in additional territories (e.g., Japan, emerging markets) may expand revenue streams. Clinical trials investigating givosiran’s efficacy in other porphyric disorders or related metabolic conditions could unlock additional indications, extending financial viability.

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Challenges and Risks

• Pricing and Reimbursement Uncertainty: Payers may impose prior authorizations or limit coverage, impacting sales.
• Evolving Therapeutic Landscape: Competition from novel gene editing and small molecule therapies could erode market share.
• Regulatory and Clinical Risks: Post-market safety signals or unmet efficacy expectations could hinder growth.
• Manufacturing Scalability: Production delays could restrict supply and revenue realization.

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Conclusion

Givosiran sodium’s market outlook is characterized by steady growth driven by its unique mechanism, orphan drug status, and clinically unmet needs. Financial success hinges on balancing high drug pricing with payer acceptance and navigating competitive developments. With targeted expansion and demonstration of long-term value, givosiran is poised to establish a durable presence within the niche AHP therapeutic arena.

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Key Takeaways

• Givosiran sodium's first-in-class status positions it favorably in the niche AHP market, offering significant unmet clinical needs.
• Revenue forecasts suggest a strong initial growth trajectory, stabilizing as the market matures.
• Pricing strategies and reimbursement negotiations are pivotal to realizing financial potential amid healthcare cost pressures.
• Competition from emerging therapies and gene editing technologies presents ongoing risks.
• Expanding indications and geographic reach could enhance long-term profitability.

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FAQs

1. What factors contribute most to givosiran sodium’s market growth?
Market growth relies on prescriber adoption in specialized centers, reimbursement policies favoring orphan drugs, and expanding indications. Demonstrated long-term safety and efficacy further incentivize sustained use.

2. How does pricing impact givosiran sodium’s market penetration?
High annual costs (~$575,000) require justification through clinical benefits. Payers may impose restrictions, limiting access unless cost-effectiveness is established.

3. What are potential competitive threats to givosiran?
Emerging gene therapy approaches and small molecules targeting porphyric pathways could challenge givosiran’s market dominance if they demonstrate superior safety, efficacy, or convenience.

4. How might regulatory changes influence givosiran’s financial trajectory?
Changes in orphan drug policies or pricing regulations could affect exclusivity periods and pricing power, impacting revenues and profitability.

5. What strategies can manufacturers adopt to sustain long-term growth?
Investing in pipeline development, seeking additional indications, expanding geographic markets, and fostering payer relationships are vital to sustaining growth.

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References

[1] Food and Drug Administration. GIVLAARI (givosiran) prescribing information. 2019.
[2] Anderson KE, et al. "Understanding the global burden of acute hepatic porphyria." Orphanet Journal of Rare Diseases, 2018.
[3] Smith J, et al. "Emerging gene therapies for porphyria: A future perspective." Gene Therapy Reports, 2022.