Alpha-1-proteinase inhibitor (human) - Biologic Drug Details

Summary for alpha-1-proteinase inhibitor (human)

Tradenames:	4
High Confidence Patents:	0
Applicants:	3
BLAs:	4
Suppliers: see list	4
Recent Clinical Trials:	See clinical trials for alpha-1-proteinase inhibitor (human)

Recent Clinical Trials for alpha-1-proteinase inhibitor (human)

Identify potential brand extensions & biosimilar entrants

Sponsor	Phase
Inhibrx, Inc.	Phase 2
Ain Shams University	PHASE3
University of Dundee	Phase 4

See all alpha-1-proteinase inhibitor (human) clinical trials

Pharmacology for alpha-1-proteinase inhibitor (human)

Mechanism of Action	Trypsin Inhibitors
Established Pharmacologic Class	Human alpha-1 Proteinase Inhibitor

Note on Biologic Patents

Matching patents to biologic drugs is far more complicated than for small-molecule drugs.

DrugPatentWatch employs three methods to identify biologic patents:

1. Brand-side disclosures in response to biosimilar applications

These patents were identified from disclosures by the brand-side company, in response to a potential biosimilar seeking to launch. They have a high certainty of blocking biosimilar entry. The expiration dates listed are not estimates — they're expiration dates as indicated by the brand-side company.

2. DrugPatentWatch analysis and brand-side disclosures

These patents were identified from searching drug labels and other general disclosures from the brand-side company. This list may exclude some of the patents which block biosimilar launch, and some of these patents listed may not actually block biosimilar launch. The expiration dates listed for these patents are estimates, based on the grant date of the patent.

3. Patents from broad patent text search

For completeness, these patents were identified by searching the patent literature for mentions of the branded or ingredient name of the drug. Some of these patents protect the original drug, whereas others may protect follow-on inventions or even inventions casually mentioning the drug. The expiration dates listed for these patents are estimates, based on the grant date of the patent.

1) High Certainty: US Patents for alpha-1-proteinase inhibitor (human) Derived from Brand-Side Litigation

No patents found based on brand-side litigation

2) High Certainty: US Patents for alpha-1-proteinase inhibitor (human) Derived from DrugPatentWatch Analysis and Company Disclosures

These patents were obtained from company disclosures

Applicant	Tradename	Biologic Ingredient	Dosage Form	BLA	Patent No.	Estimated Patent Expiration	Source
Takeda Pharmaceuticals U.s.a., Inc.	ARALAST, ARALAST NP	alpha-1-proteinase inhibitor (human)	For Injection	125039	5,616,693	2016-07-01	DrugPatentWatch analysis and company disclosures
Takeda Pharmaceuticals U.s.a., Inc.	ARALAST, ARALAST NP	alpha-1-proteinase inhibitor (human)	For Injection	125039	7,807,435	2025-08-11	DrugPatentWatch analysis and company disclosures
Csl Behring Llc	ZEMAIRA	alpha-1-proteinase inhibitor (human)	For Injection	125078	10,098,950	2037-09-15	DrugPatentWatch analysis and company disclosures
Csl Behring Llc	ZEMAIRA	alpha-1-proteinase inhibitor (human)	For Injection	125078	10,179,183	2037-05-30	DrugPatentWatch analysis and company disclosures
Csl Behring Llc	ZEMAIRA	alpha-1-proteinase inhibitor (human)	For Injection	125078	10,179,184	2034-11-25	DrugPatentWatch analysis and company disclosures

3) Low Certainty: US Patents for alpha-1-proteinase inhibitor (human) Derived from Patent Text Search

These patents were obtained by searching patent claims

Applicant	Tradename	Biologic Ingredient	Dosage Form	BLA	Patent No.	Estimated Patent Expiration	Source

International Patents for alpha-1-proteinase inhibitor (human)

Country	Patent Number	Estimated Expiration
Spain	2625792
Australia	2010219370
Norway	20200595
Russian Federation	2571929
Lithuania	3165539

Supplementary Protection Certificates for alpha-1-proteinase inhibitor (human)

Supplementary Protection Certificate	SPC Country	SPC Expiration	SPC Description
300919	Netherlands		PRODUCT NAME: BRODALUMAB; REGISTRATION NO/DATE: EU/1/16/1155/001-002 20170719
C 2018 002	Romania		PRODUCT NAME: BRODALUMAB; NATIONAL AUTHORISATION NUMBER: EU/1/16/1155/001-002; DATE OF NATIONAL AUTHORISATION: 20170717; NUMBER OF FIRST AUTHORISATION IN EUROPEAN ECONOMIC AREA (EEA): EU/1/16/1155/001-002; DATE OF FIRST AUTHORISATION IN EEA: 20170717
2/2018	Austria		PRODUCT NAME: "BRODALUMAB"; REGISTRATION NO/DATE: EU/1/16/1155/001-002 (MITTEILUNG) 20170719
CA 2017 00063	Denmark		PRODUCT NAME: ISOLERET ANTISTOF ELLER FRAGMENT DERAF OMFATTENDE LET KAEDE OG TUNG KAEDE CDR-SEKVENSER IFOELGE EP-B1-2076541 KRAV 1 (SEQ ID NO: 224, 225, 226 OG 146, 147, 148).....; REG. NO/DATE: EU/1/16/1155/001-002 20170719
CR 2017 00063	Denmark		PRODUCT NAME: BRODALUMAB; REG. NO/DATE: EU/1/16/1155/001-002 20170719

Market Dynamics and Financial Trajectory for Alpha-1-Proteinase Inhibitor (Human)

Last updated: August 18, 2025

Introduction

Alpha-1-proteinase inhibitor (human), commercially known as alpha-1 antitrypsin (AAT), is a biologic drug primarily used for treating Alpha-1 Antitrypsin Deficiency (AATD)—a hereditary disorder that can lead to chronic obstructive pulmonary disease (COPD) and liver disease. The drug functions by supplementing deficient alpha-1 antitrypsin levels, thereby reducing lung tissue damage caused by unchecked neutrophil elastase activity. Understanding current market dynamics and financial trajectories of this biologic offers critical insight for stakeholders navigating from manufacturing to commercialization.

Market Landscape and Drivers

Growing Prevalence and Unmet Medical Need

AATD affects approximately 1 in 2,500 to 5,000 individuals of European descent, with many cases remaining undiagnosed due to limited awareness and testing (1). The rising prevalence, especially in aging populations, amplifies demand for AAT replacement therapies. Furthermore, the disease burden, with progressive lung damage and liver failure, underscores the urgent need for effective therapeutics.

Regulatory and Reimbursement Environment

European and U.S. regulatory bodies have approved several formulations of alpha-1-proteinase inhibitor, including both pooled plasma-derived and recombinant versions. The approval landscape continues to evolve, with expanded indications and improved formulations driving market growth (2). Reimbursement policies vary but are increasingly favorable for genetically targeted biologics, provided robust clinical and economic data.

Innovations and Pipeline Developments

Current formulations, such as Prolastin® and Zemaira®, primarily derived from human plasma, face competition from biosimilars and recombinant variants. Emerging efforts aim to develop longer-lasting formulations, subcutaneous delivery options, and gene therapy approaches, potentially disrupting current market paradigms (3).

Market Challenges and Restraints

Supply Chain and Ethical Concerns

Plasma-derived products are subject to supply constraints due to limited donor pools. Ethical considerations surrounding plasma collection and safety risks associated with pathogen transmission necessitate rigorous screening, increasing manufacturing costs.

High Production Costs and Pricing Pressures

Biologics, especially plasma-derived products, entail significant manufacturing costs, impacting pricing strategies. Payers and healthcare systems are scrutinizing high-cost biologics, leading to reimbursement challenges and calls for more cost-effective alternatives.

Market Penetration Limitations

Despite the growing demand, disease underdiagnosis hampers market expansion. Limited awareness campaigns and variability in physician familiarity with AATD restrict timely diagnosis and treatment initiation.

Financial Trajectory and Revenue Projections

Historical Revenue Trends

The alpha-1-antitrypsin market has demonstrated steady growth over the past decade. Global sales of plasma-derived AAT products exceeded $1.3 billion in 2022, driven predominantly by North America and Europe (4). The primary players—CSL Behring, Alexion (a subsidiary of AstraZeneca), and Grifols—control a significant market share through established manufacturing infrastructure and broad distribution networks.

Forecast Growth and Drivers

Analysts project compounded annual growth rates (CAGR) of approximately 4-6% from 2023 to 2030, driven by:

• Expanding Patient Population: Enhanced screening programs and increased awareness are expected to elevate diagnosed cases, fueling sales.
• Product Innovation: Introduction of long-acting formulations and subcutaneous delivery devices can improve patient compliance, opening new revenue streams.
• Pipeline Progress: Successful clinical trials of recombinant AAT and gene therapies may reshape revenue landscapes, offering potentially lower-cost, scalable solutions.

Impact of Biosimilars and Alternative Therapies

Biosimilar entrants could exert downward pressure on prices, which might temper revenue growth post-2025. Conversely, differentiation through enhanced efficacy or delivery mechanisms can sustain premium pricing for innovative formulations.

Market Penetration in Emerging Economies

Emerging markets, including Asia-Pacific, present opportunities for growth, contingent on establishing reliable plasma collection and distribution channels, and navigating regulatory landscapes.

Strategic Market Opportunities

• Personalized Medicine: Genetic screening to identify candidates with severe AATD can optimize treatment outcomes and resource utilization.
• Combination Therapies: Investigating synergistic regimens incorporating anti-inflammatory agents could enhance therapeutic efficacy.
• Gene Therapy Prospects: Restorative approaches, currently in preclinical and clinical stages, could, if commercialized effectively, drastically shift the financial trajectory, potentially reducing long-term treatment costs.

Risks and Uncertainties

• Regulatory Delays and Approvals: New formulations or biosimilars face rigorous approval processes, potentially delaying market entry.
• Supply Constraints: Plasma procurement bottlenecks or safety incidents could disrupt supply, impacting revenues.
• Pricing and Reimbursement Policies: Increasing cost containment measures may limit revenue growth, especially in healthcare systems emphasizing value-based care.

Conclusion

The market for alpha-1-proteinase inhibitor (human) remains characterized by steady demand driven by clinical need, but faces headwinds from high production costs, supply constraints, and competitive innovations. Financial trajectories suggest moderate growth, with an optimistic outlook conditioned on advancements in recombinant technology, gene therapy, and novel delivery methods. Stakeholders that navigate these dynamics effectively—by fostering innovation, expanding into emerging markets, and engaging with payers—can capitalize on the evolving landscape.

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Key Takeaways

• The global alpha-1-antitrypsin market is projected to grow at a CAGR of 4-6% toward 2030, driven by increasing diagnostic rates and product innovations.
• Supply chain sustainability and ethical plasma collection are critical for maintaining market stability.
• Emerging recombinant and gene therapies, alongside biosimilars, present both growth avenues and competitive pressures.
• Reimbursement policies and cost containment efforts will significantly influence financial profitability.
• Strategic investments in personalized medicine and expanding into emerging markets can maximize revenue potential.

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FAQs

1. What factors are most influencing the growth of alpha-1-proteinase inhibitor (human) markets?
   Growth is primarily driven by increasing prevalence of diagnosed AATD cases, advancements in biologic formulations, and expanding awareness campaigns. Innovation in delivery methods and pipeline development further catalyze market expansion.

2. How do biosimilars impact the alpha-1-antitrypsin market?
   Biosimilars can introduce competitive pricing, potentially reducing revenue. However, differentiation through enhanced efficacy or delivery can mitigate pricing pressures and preserve market share.

3. What is the role of gene therapy in the future of alpha-1-proteinase inhibitor treatment?
   Gene therapy offers a potentially transformative approach by restoring endogenous AAT production, which may reduce or eliminate the need for exogenous replacement, reshaping the financial landscape if approved at scale.

4. Are emerging markets a significant opportunity for alpha-1-antitrypsin therapeutics?
   Yes. While current markets are concentrated primarily in North America and Europe, emerging economies offer growth opportunities contingent on improving plasma collection, regulatory navigation, and healthcare infrastructure.

5. What are the main challenges in sustaining profitability for biologic manufacturers of alpha-1-proteinase inhibitor?
   Challenges include high manufacturing costs, supply chain constraints, regulatory hurdles, and pricing pressures from payers aiming to control costs without compromising patient access.

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References

[1] Stoller, J. K., & Aboussouan, L. S. (2012). Alpha1-antitrypsin deficiency. The Lancet, 379(9824), 2093–2101.

[2] European Medicines Agency. (2021). Summary of Opinion (CHMP) for alpha-1-proteinase inhibitor products.

[3] Pagnamenta, A. (2020). Recombinant Alpha-1-Antitrypsin: The Future of Treatment? Nature Biotechnology.

[4] MarketWatch. (2023). Global alpha-1-antitrypsin market size and forecast.