givosiran sodium - Profile

What are the generic 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 eight patents protecting this compound. Additional information is available in the individual branded drug profile pages.

Givosiran sodium has one hundred and eighty-two patent family members in forty-three countries.

Summary for givosiran sodium

International Patents:	182
US Patents:	8
Tradenames:	1
Applicants:	1
NDAs:	1
Patent Litigation and PTAB cases:	See patent lawsuits and PTAB cases for givosiran sodium

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

Constraining patent/regulatory exclusivity:

INDICATED FOR THE TREATMENT OF ADULTS WITH ACUTE HEPATIC PORPHYRIA (AHP)

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.

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
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 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
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 givosiran sodium

Country	Patent Number	Title	Estimated Expiration
Australia	2020202970	COMPOSITIONS AND METHODS FOR INHIBITING EXPRESSION OF THE ALAS1 GENE
Japan	2019022519
Japan	2015518373	ＡＬＡＳ１遺伝子の発現を阻害する組成物および方法

Supplementary Protection Certificates for givosiran sodium

Patent Number	Supplementary Protection Certificate	SPC Country	SPC Expiration	SPC Description
3052628	C20200026 00361	Estonia		PRODUCT NAME: GIVOSIRAAN;REG NO/DATE: EU/1/20/1428 04.03.2020
3052628	2020/038	Ireland		PRODUCT NAME: GIVOSIRAN OR A PHARMACEUTICALLY ACCEPTABLE SALT THEREOF; NAT REGISTRATION NO/DATE: EU/1/20/1428 20200304; FIRST REGISTRATION NO/DATE: EU/1/20/1428 04/03/2020
3052628	122020000045	Germany		PRODUCT NAME: GIVOSIRAN ODER EIN PHARMAZEUTISCH VERTRAEGLICHES SALZ DAVON; REGISTRATION NO/DATE: EU/1/20/1428 20200302

Givosiran Sodium: Investment Scenario, Market Dynamics, and Financial Trajectory

Last updated: February 3, 2026

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Summary

Givosiran sodium (brand name Givlaari) is a liver-targeted RNA interference (RNAi) therapeutic approved by the FDA in 2019 for the treatment of acute hepatic porphyria (AHP). Its innovative mechanism of action, targeting the hepatic enzyme ALAS1, positions it as a first-in-class therapy with substantial commercial potential amid the rare disease and genetic disorder markets. This report analyzes the current market landscape, investment opportunities, competitive environment, and the financial trajectory for Givosiran sodium, integrating recent developments, regulatory factors, and market outlooks.

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What is the Current Market Position of Givosiran Sodium?

Aspect	Details
Approval Date	November 2019 (FDA)
Indication	Acute hepatic porphyria (AHP)
Administration	Subcutaneous injection
Market Launch	2020
Key Competitors	No direct RNAi competitors; limited therapeutics specific to AHP
Current Sales (2022)	Approx. $150 million globally
Market Penetration	Estimated 25% of eligible patients (market size: 4,500–6,000 in US and EU)

Source: [1], [2]

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Market Dynamics: Analyzing the Drivers and Challenges

1. Market Size and Patient Population

Region	Estimated AHP Patients	Market Penetration	Potential Revenue
US	3,000–4,000	25%	~$150 million
EU	1,500–2,500	15%	~$60 million
ROW (Rest of World)	1,000	Limited	<$10 million

Implication: The addressable market remains underpenetrated owing to diagnostic challenges and limited awareness, offering upside with improved detection.

2. Competitive Landscape and Market Entry Barriers

Competitors	Status
Hematology/Genetics	No FDA-approved alternatives for AHP
Other RNAi therapies	No direct competition, but emerging pipeline in genetic rare diseases
Symptomatic Treatments	Acute symptom management drugs, no disease-controlling agents

Barrier Factors:

• Orphan drug designation facilitates market exclusivity until 2024–2027 in key regions.
• High development costs and regulatory hurdles deter new entrants.
• Limited patient pool reduces immediate profitability, making large-scale investments risky.

3. Reimbursement and Pricing Environment

Policy Aspect	Details
Pricing (US)	Approx. $575,000/year per patient
Reimbursement	CMS and private insurers generally cover Givosiran for eligible patients
Pricing Challenges	Cost-effectiveness concerns due to high treatment price; ongoing payer negotiations

Outcome: Reimbursement policies heavily influence market adoption; payers’ willingness to pay hinges on clinical and economic evidence.

4. Regulatory and Patent Landscape

Regulatory & Patent Elements	Details
Orphan Drug Status	Granted in US and EU
Patent Portfolio	Initially secured via Alnylam’s RNAi platform; patents expiring between 2030–2035
Regulatory Exclusivity	7–12 years post-approval depending on jurisdiction

Implication: Patent disputes could influence future licensing, licensing negotiations, or generic entry.

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Financial Trajectory Analysis

1. Revenue Projections (2023–2028)

Year	Revenue ($ millions)	Assumptions
2023	$180 million	20% YoY growth, increased market penetration
2024	$220 million	Expanded diagnosis, broader access
2025	$280 million	New geographic launches, possible expanded indications
2026	$340 million	Mature market dynamics
2027	$400 million	Penetration plateau, additional pricing leverage
2028	$450 million	New patient identification, potential label expansion

Sources: Company filings, industry estimates, market research reports.

2. Cost Structure & Profitability

Cost Component	Details
R&D Expenses	Approximately 35–40% of revenue
Manufacturing Costs	Approximately 15–20% of revenue
Sales & Marketing	~25% of revenue
Operating Margin	Estimated 20–25% in mature years

Note: Significant investments in pharmacovigilance, further clinical trials, and market expansion are anticipated.

3. Investment Considerations

Aspect	Details
Growth Potential	High among rare disease therapies, especially with label expansion
Risk Factors	Market penetration slowdowns, payer resistance, patent expirations, emerging pipeline
Partnerships & Licensing	Strategic alliances with biopharma incumbents could mitigate risk

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Comparison With Similar Therapies and Market Trends

Therapy	Disease	Approval Year	Market Size	Revenue (2022)	Key Differentiators
Givosiran	AHP	2019	~$150 million	Approx. $150 million	First RNAi for AHP, high unmet need
Inotersen (Tegsedi)	Hereditary ATTR amyloidosis	2018	$300 million	Growing	Different indications, similar RNAi platform
Patisiran (Onpattro)	Hereditary ATTR amyloidosis	2018	$460 million	Growing	Broader lipid delivery

Insight: Givosiran benefits from being first-in-class with limited direct competition, yet future pipeline entrants could alter dynamics.

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Strategic Investment Outlook

Factor	Impact
Market Penetration Growth	High potential with increased diagnosis and awareness
Regulatory Approvals	Label expansion to other porphyrias or genetic disorders would enhance market size
Partnerships & Collaborations	Could accelerate commercialization and access
Patent and Exclusivity	Maintains market dominance until late 2020s

Overall: Givosiran sodium presents an attractive long-term investment risk-reward for stakeholders familiar with rare disease therapeutics. Its market is poised for growth, driven by increasing diagnosis, better reimbursement negotiability, and potential new indications.

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Key Takeaways

• Market Opportunity: The global AHP market remains underpenetrated with room for significant expansion, driven by better diagnosis and broader awareness.
• Revenue Growth: Projected CAGR of approximately 22-25% through 2028, assuming steady market penetration and additional geographic launches.
• Competitive Environment: Currently limited, with no direct RNAi competitors; however, pipeline development in rare disease space warrants continual watch.
• Pricing & Reimbursement: High-cost pricing strategy is supported by orphan drug status and demonstrated clinical benefits; negotiations will remain pivotal.
• Future Prospects: Label expansions, combination therapies, and new indications could redefine Givosiran’s market trajectory, bolstering its financial outlook.

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FAQs

1. What are the main factors influencing Givosiran’s market expansion?
Improved diagnostics, increased awareness among rare disease communities, positive payer coverage decisions, and potential label extensions into related porphyria subtypes.

2. How does Givosiran compare to other RNAi therapies in development?
While it is a first-in-class approved therapy for AHP, pipeline contenders target similar or related conditions, with advancements in delivery technologies and broader indications.

3. What are the key patent and exclusivity considerations for Givosiran?
Patent protections extend into the early 2030s, with regulatory exclusivity ranging from 7–12 years, ensuring some market protection against generics and biosimilars.

4. How might pricing dynamics evolve in the future?
Pressure from payers and health authorities could lead to negotiated discounts or value-based pricing, impacting revenue growth but potentially increasing market access.

5. What are the primary risks associated with investing in Givosiran?
Market penetration delays, emerging competitors, regulatory changes, patent cliffs, and potential safety concerns impacting uptake.

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References

[1] Alnylam Pharmaceuticals. (2022). Givlaari (Givosiran) Prescribing Information.
[2] IQVIA. (2022). Rare Disease Market Report.
[3] FDA. (2019). Givlaari (Givosiran) Approval Announcement.
[4] Market Research Future. (2022). RNAi Therapeutics Market Overview.
[5] EvaluatePharma. (2022). 2022 Global Pharma Revenue Database.