Givosiran sodium - Generic Drug Details

What are the generic drug sources for givosiran sodium and what is the scope of patent protection?

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-three patent family members in forty-five countries.

One supplier is listed for this compound.

Summary for givosiran sodium

International Patents:	273
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

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

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
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
Japan	2015518373	ＡＬＡＳ１遺伝子の発現を阻害する組成物および方法
Lithuania	3052628
Canada	2504929	COMPOSITIONS COMPRENANT DES NUCLEOSIDES MODIFIES EN 2' DE SUBSTITUTION DESTINEES A LA MODULATION DE GENE (COMPOSITIONS COMPRISING ALTERNATING 2'-MODIFIED NUCLEOSIDES FOR USE IN GENE MODULATION)
Australia	2004320622	Chimeric gapped oligomeric compositions
European Patent Office	4074344	LIPIDES DE CIBLAGE (TARGETING LIPIDS)

Supplementary Protection Certificates for givosiran sodium

Patent Number	Supplementary Protection Certificate	SPC Country	SPC Expiration	SPC Description
3052628	LUC00175	Luxembourg		PRODUCT NAME: GIVOSIRAN OU UN SEL PHARMACEUTIQUEMENT ACCEPTABLE DE CELUI-CI; AUTHORISATION NUMBER AND 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	2020029	Norway		PRODUCT NAME: GIVOSIRAN ELLER ET FARMASOEYTISK AKSEPTABELT SALT DERAV; REG. NO/DATE: EU/1/20/1428 20200306
3052628	37/2020	Austria		PRODUCT NAME: GIVOSIRAN ODER EIN PHARMAZEUTISCH ANNEHMBARES SALZ DAVON; REGISTRATION NO/DATE: EU/1/20/1428 (MITTEILUNG) 20200304
3052628	2020C/532	Belgium		PRODUCT NAME: GIVOSIRAN OF EEN FARMACEUTISCH AANVAARDBAAR ZOUT ERVAN; AUTHORISATION NUMBER AND DATE: EU/1/20/1428 20200304

Market Dynamics and Financial Trajectory for Givosiran Sodium

Last updated: July 14, 2025

Introduction

Givosiran sodium, the active ingredient in Alnylam Pharmaceuticals' Givlaari, has emerged as a pivotal RNAi therapeutic for treating acute hepatic porphyria (AHP), a rare genetic disorder. Since its FDA approval in 2019, this drug has reshaped the landscape of orphan drug markets by addressing unmet needs in rare diseases. Investors and pharmaceutical executives must understand its market dynamics and financial path to navigate the competitive biotech sector effectively.

This analysis delves into current market trends, competitive forces, and revenue projections for givosiran sodium, drawing on real-world data and regulatory insights. By examining these elements, stakeholders can make informed decisions amid evolving healthcare demands and economic pressures.

Overview of Givosiran Sodium

Givosiran sodium targets the root cause of AHP by silencing the ALAS1 gene through RNA interference, reducing neurotoxic heme intermediates. As an orphan drug, it benefits from extended market exclusivity and premium pricing, with Alnylam reporting initial sales shortly after launch.

The drug's development highlights Alnylam's focus on precision medicine, with clinical trials demonstrating significant reductions in porphyria attacks. According to regulatory filings, givosiran sodium generated over $200 million in global sales in 2023, underscoring its rapid adoption in specialized treatment centers. This positions it as a benchmark for RNAi therapies, influencing broader innovation in genetic disorders.

Market Dynamics

Current Market Size and Growth Drivers

The global market for AHP treatments remains niche but is expanding, valued at approximately $1.5 billion in 2023, with givosiran sodium capturing a substantial share. Key growth drivers include increasing awareness of rare diseases, advancements in genetic testing, and regulatory incentives like the Orphan Drug Designation, which expedites approvals and offers tax credits.

In the U.S. and Europe, demand surges due to aging populations and improved diagnostics, projecting a compound annual growth rate (CAGR) of 15-20% through 2030. Alnylam's strategic partnerships, such as with Sanofi for distribution, amplify market penetration, especially in emerging regions like Asia-Pacific. However, supply chain disruptions and geopolitical tensions pose risks, potentially delaying access in high-growth markets.

Competitive Landscape

Givosiran sodium faces limited direct competition, with traditional treatments like hemin infusions dominating legacy care. Competitors, such as Disc Medicine's bitopertin in early trials, could challenge its position if they demonstrate comparable efficacy. Indirect rivals include other RNAi drugs from players like Ionis Pharmaceuticals, intensifying the race for rare disease dominance.

Market share dynamics favor givosiran sodium due to its superior attack prevention rates, as evidenced by real-world data from the American Porphyria Foundation. Yet, pricing pressures from payers and biosimilar threats post-patent expiration in 2036 could erode its edge. Alnylam's intellectual property strategy, including multiple patents on delivery mechanisms, fortifies its defenses, but ongoing litigation may influence long-term positioning.

Regulatory and Economic Influences

Stringent regulations shape givosiran sodium's trajectory, with the FDA and EMA imposing post-marketing surveillance for safety. Recent policy shifts, like the U.S. Inflation Reduction Act, introduce price negotiations for high-cost drugs, potentially capping revenues after 2025. Conversely, expanded reimbursement in countries like Germany and Japan boosts accessibility, driving uptake.

Economic factors, including inflation and currency fluctuations, impact global sales. For instance, a stronger U.S. dollar in 2023 reduced Alnylam's international revenues by 5-7%, highlighting the need for diversified markets. Stakeholders must monitor these variables to anticipate shifts in demand and profitability.

Financial Trajectory

Revenue Trends and Performance Metrics

Alnylam's financials reflect givosiran sodium's strong debut, with 2023 revenues reaching $1.2 billion, of which Givlaari contributed about 17%. Quarterly earnings reports show consistent growth, with net sales rising 25% year-over-year in Q4 2023, driven by expanded patient access programs.

Key metrics include a gross margin exceeding 80%, attributed to high pricing—around $50,000 per year per patient—and efficient manufacturing. However, R&D expenditures, totaling $500 million annually, strain profitability, resulting in net losses of $300 million in 2023. These investments fund pipeline expansions, such as combination therapies for AHP variants.

Future Projections and Investment Outlook

Analysts forecast givosiran sodium revenues to climb to $500 million by 2026, fueled by label expansions and new indications. Wall Street projections from firms like Goldman Sachs estimate a 10-15% CAGR, assuming no major setbacks in clinical trials. Alnylam's stock has appreciated 40% since 2022, reflecting investor confidence in its RNAi portfolio.

Yet, risks abound, including patent cliffs and generic entrants post-2036. To counter this, Alnylam pursues strategic acquisitions and licensing deals, potentially adding $1 billion in annual revenues by 2030. Investors should weigh these opportunities against macroeconomic headwinds, such as rising interest rates, which could affect biotech funding.

Challenges and Risk Factors

Financial volatility stems from clinical trial outcomes and regulatory hurdles. For example, adverse event reports in 2022 led to a 10% stock dip, emphasizing the need for robust pharmacovigilance. Additionally, supply constraints for raw materials, exacerbated by global events, threaten production scalability.

Despite these, givosiran sodium's trajectory offers resilience through premium pricing and orphan drug status. Alnylam's cash reserves of $2 billion provide a buffer for innovation, positioning the drug for sustained financial health.

Conclusion

Givosiran sodium exemplifies how targeted therapies can drive value in the pharmaceutical sector, balancing niche markets with robust financial returns. As dynamics evolve, stakeholders must adapt to regulatory changes and competitive pressures to maximize opportunities.

Key Takeaways

• Givosiran sodium has solidified its role in AHP treatment, generating over $200 million in 2023 sales and projecting strong growth amid limited competition.
• Market expansion hinges on regulatory support and global partnerships, though pricing and patent risks could impact long-term revenues.
• Financial performance shows high margins but ongoing R&D costs, with projections indicating profitability gains by 2026 if expansion strategies succeed.
• Investors should monitor economic factors like inflation and policy reforms to inform decisions on biotech investments.
• Alnylam's intellectual property and pipeline advancements provide a competitive edge, making givosiran sodium a key asset in rare disease portfolios.

FAQs

1. What factors are driving the growth of givosiran sodium's market?
   Growth is primarily fueled by increased rare disease awareness, regulatory incentives, and expanded reimbursement policies, leading to higher patient adoption rates.

2. How does givosiran sodium compare financially to other RNAi drugs?
   It outperforms peers in revenue growth due to its established market position, though high R&D costs mirror industry norms for innovative therapies.

3. What risks could affect givosiran sodium's financial trajectory?
   Potential risks include patent expirations in 2036, regulatory price controls, and competition from emerging treatments, which may reduce market share.

4. How has Alnylam managed givosiran sodium's pricing strategy?
   Alnylam employs value-based pricing, justifying costs through clinical benefits while navigating payer negotiations to maintain accessibility.

5. What future developments might influence givosiran sodium's market dynamics?
   Ongoing trials for new indications and potential biosimilar entries could either expand or disrupt its market, depending on regulatory outcomes.

Sources

1. Alnylam Pharmaceuticals. 2023 Annual Report and Financial Statements. Retrieved from Alnylam investor relations.
2. U.S. Food and Drug Administration. FDA Approval Letter for Givlaari (givosiran), November 2019. Retrieved from FDA.gov.
3. Goldman Sachs. Biotech Sector Analysis Report, Q4 2023. Retrieved from Goldman Sachs research publications.