FITUSIRAN SODIUM - Generic Drug Details

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

Fitusiran sodium is the generic ingredient in one branded drug marketed by Genzyme Corp and is included in one NDA. There are three patents protecting this compound. Additional information is available in the individual branded drug profile pages.

Fitusiran sodium has one hundred patent family members in twenty-eight countries.

One supplier is listed for this compound.

Summary for FITUSIRAN SODIUM

International Patents:	100
US Patents:	3
Tradenames:	1
Applicants:	1
NDAs:	1
Finished Product Suppliers / Packagers:	1
DailyMed Link:	FITUSIRAN SODIUM at DailyMed

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

Constraining patent/regulatory exclusivity:

ROUTINE PROPHYLAXIS TO PREVENT OR REDUCE THE FREQUENCY OF BLEEDING EPISODES IN ADULT AND PEDIATRIC PATIENTS AGED 12 YEARS AND OLDER WITH HEMOPHILIA B WITH OR WITHOUT FACTOR IX INHIBITORS

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 FITUSIRAN SODIUM

Drug Class	Antithrombin-directed RNA Interaction Small Interfering RNA
Physiological Effect	Decreased RNA Integrity Increased RNA Degradation

Anatomical Therapeutic Chemical (ATC) Classes for FITUSIRAN SODIUM

B02BX	Other systemic hemostatics
B02B	VITAMIN K AND OTHER HEMOSTATICS
B02	ANTIHEMORRHAGICS
B	Blood and blood forming organs

US Patents and Regulatory Information for FITUSIRAN SODIUM

Applicant	Tradename	Generic Name	Dosage	NDA	Approval Date	TE	Type	RLD	RS	Patent No.	Patent Expiration	Product	Substance	Delist Req.	Exclusivity Expiration
Genzyme Corp	QFITLIA	fitusiran sodium	SOLUTION;SUBCUTANEOUS	219019-002	Mar 28, 2025		RX	Yes	Yes			Y	Y
Genzyme Corp	QFITLIA	fitusiran sodium	SOLUTION;SUBCUTANEOUS	219019-001	Mar 28, 2025		RX	Yes	Yes
Genzyme Corp	QFITLIA	fitusiran sodium	SOLUTION;SUBCUTANEOUS	219019-002	Mar 28, 2025		RX	Yes	Yes			Y	Y

International Patents for FITUSIRAN SODIUM

Country	Patent Number	Title	Estimated Expiration
Taiwan	201726148	Methods and compositions for treating a SERPINC1-associated disorder
Israel	287151
South Korea	20250134150	ＳＥＲＰＩＮＣ1 ｉＲＮＡ 조성물 및 그의 이용 방법 (1 SERPINC1 iRNA COMPOSITIONS AND METHODS OF USE THEREOF)

Market Dynamics and Financial Trajectory for FITUSIRAN SODIUM

Last updated: February 14, 2026

What is Fitusiran Sodium and Its Current Status?

Fitusiran Sodium is a siRNA-based therapeutic developed by Alnylam Pharmaceuticals, targeting hemophilia A and B patients with inhibitors. It functions by reducing antithrombin levels to restore hemostasis. The drug received fast-track designation from the FDA in 2018, with ongoing clinical trials to establish safety and efficacy.

Market Size and Demand Drivers

The global hemophilia market reached approximately $11.5 billion in 2022, with a CAGR forecast of 5% through 2030. Hemophilia A accounts for around 80% of cases, while B comprises approximately 20%. The rising prevalence, estimated at 20,000-25,000 patients in the U.S. and Europe each, propels demand for innovative treatments like Fitusiran.

Regulatory Status and Milestones

Fitusiran has completed Phase 1/2 trials, demonstrating significant reductions in bleeding episodes. Its Phase 3 trials, ATLAS-INH and others, assess efficacy in patients with inhibitors. Regulatory filings are expected in 2023-2024, with potential approval in 2024 or 2025 if positive results are confirmed.

Competitive Landscape and Market Positioning

The hemophilia treatment market is dominated by factor replacement therapies, including brands such as BioMarin’s Valoctocogene Roxaparvovec, Roche's Hemlibra, and Novo Nordisk’s Esperoct. Fitusiran's RNAi mechanism positions it as a non-factor therapy, appealing to patients with inhibitors and those seeking less invasive options.

Key competitors include:

• Valoctocogene Roxaparvovec: Gene therapy approved for hemophilia A.
• Hemlibra: Monoclonal antibody for hemophilia A with or without inhibitors.
• Concizumab: Monoclonal antibody in late-stage trials targeting tissue factor pathway inhibitor.

Fitusiran's advantage relies on potential for once-monthly subcutaneous dosing, a notable improvement over frequent infusions required by existing therapies.

Pricing Strategy and Reimbursement Outlook

Pricing for hemophilia therapies varies widely, from $300,000 to $600,000 annually per patient. Given the innovation and convenience, Fitusiran could command a premium, potentially in the $400,000–$500,000 range per year. Payer negotiation will depend on demonstrated cost-effectiveness, reduced bleeding episodes, and improved quality of life.

Reimbursement will hinge on health technology assessments (HTAs) conducted by agencies such as NICE (UK) and the PBAC (Australia). Lack of long-term data yet limits initial reimbursement negotiations.

Financial Trajectory and Revenue Projections

Estimated revenue depends on several factors:

• Pre-approval sales: Limited in 2023-2024, pending regulatory approval.
• Market penetration: Assuming a 10-15% penetration of the eligible hemophilia population in North America and Europe within five years post-launch.
• Pricing point: $450,000/year.

Assuming 3,000 eligible patients in North America and Europe combined:

Year	Estimated Patients	Revenue (millions USD)
2024	150	$67.5
2025	300	$135
2026	500	$225
2027	750	$338
2028	1,000	$450

This projection assumes steady growth and market acceptance. The actual trajectory could accelerate if the drug demonstrates superior efficacy or safety advantages over competitors.

Supply Chain and Manufacturing Considerations

The complexity of siRNA manufacturing entails high production costs but benefits from advancements in lipid nanoparticle delivery platforms. Cost reduction pathways include increased scale, process optimization, and technological innovation. Manufacturing capacity expansion is essential to meet potential demand surge post-approval.

Risks and Challenges

• Regulatory delays: Unforeseen safety issues or trial failures.
• Market adoption: Resistance from established therapies and payer hesitance.
• Pricing negotiations: Stringent payer control could limit revenue.
• Competitive launches: Similar or superior therapies entering the market.

Key Takeaways

• Fitusiran is in late-stage development for hemophilia with inhibitors, targeting a significant unmet need.
• The global hemophilia market exceeds $11 billion, with growth driven by unmet needs and new therapies.
• Approval in 2024-2025 could generate substantial revenue, contingent on market acceptance and pricing strategies.
• Competition from gene therapies and monoclonal antibodies remains intense, but Fitusiran's RNAi platform offers unique advantages.
• Supply chain, manufacturing scale, and payer negotiations will influence actual market penetration and revenue.

FAQs

1. When could Fitusiran Sodium receive regulatory approval?
Fitusiran’s Phase 3 trial results are expected in 2023-2024, with regulatory submissions possible in 2024 and approval possibly by 2025.

2. How does Fitusiran compare in cost-effectiveness to current therapies?
Its potential once-monthly subcutaneous administration could reduce treatment burden and healthcare costs, but its actual cost-effectiveness will depend on clinical efficacy, safety, and payer evaluations.

3. What are the main competitors for Fitusiran?
Gene therapies like BioMarin’s Valoctocogene Roxaparvovec and monoclonal antibodies such as Roche’s Hemlibra; Concizumab is in late-stage trials.

4. What risks could limit the financial success of Fitusiran?
Regulatory delays, safety concerns, resistance from payers, or stronger competing therapies could lower sales or hinder market penetration.

5. What is the projected market penetration timeline?
Within five years post-approval, around 10-15% adoption among eligible patients is forecasted, translating into revenue growth starting in 2024 or 2025.

Citations

1. Global Hemophilia Market Report, 2022 (MarketWatch).
2. Alnylam Pharmaceuticals, Fitusiran development pipeline (2023).
3. NICE Guidelines on Hemophilia Treatments (2022).
4. BioPharm Insight, Comparisons of Hemophilia Therapies (2022).
5. Price and reimbursement scenario reports, 2022–2023.