KLKB1 Inhibitors Market Summary

• The KLKB1 Inhibitors Market Size is anticipated to grow with a significant CAGR during the study period (2020-2034).
• Plasma kallikrein inhibitors represent a promising therapeutic approach for diseases characterized by excessive vascular permeability, inflammation, and aberrant blood pressure regulation, though clinical use beyond HAE is still emerging.
• The key KLKB1 Inhibitors companies actively involved include - KalVista Pharmaceuticals, Ionis Pharmaceuticals, Takeda Pharmaceuticals, BioCryst Pharmaceuticals, Intellia Therapeutics, Astria Therapeutics, Rezolute, and others.

KLKB1 Inhibitors Market and Epidemiology Analysis

• KLKB1 inhibitors such as TAKHZYRO (lanadelumab), ORLADEYO (berotralstat), EKTERLY (sebetralstat), and DAWNZTERA (donidalorsen) have rapidly become standard-of-care therapies for hereditary angioedema (HAE), addressing both prophylaxis and acute attack treatment with multiple administration routes (oral, injectable, and RNA-based).
• The recent approval of EKTERLY as the first oral on-demand therapy and DAWNZERA as the first RNA-based prophylactic, alongside NTLA-2002 gene-editing therapy in Phase III, highlights a pipeline shift toward convenience, long-term efficacy, and potential one-time cures.
• Elevated plasma kallikrein levels are linked to increased cardiovascular risk, including myocardial infarction and stroke in men. Higher kallikrein levels have also been observed in children with abnormal lipid profiles and metabolic syndrome, correlating with key cardiovascular risk factors. These associations suggest plasma kallikrein plays a significant role in vascular inflammation and cardiovascular disease progression.
• Targeting or reducing PKal activity has been validated as a beneficial approach for managing conditions such as HAE, diabetic microvascular complications, and cardiovascular diseases.
• Investigational candidates like RZ402 in diabetic macular edema (DME) represent the first wave of KLKB1 inhibitors moving into high-prevalence indications, expanding commercial potential far beyond rare diseases like hereditary angioedema.
• In August 2025, Ionis Pharmaceuticals announced that the US Food and Drug Administration (FDA) had approved DAWNZERA for prophylaxis to prevent attacks of HAE in adult and pediatric patients 12 years of age and older. DAWNZERA is the first and only RNA-targeted medicine approved for hereditary angioedema.
• Kallikrein inhibitors are primarily used to treat hereditary angioedema, with growing investigation in diabetic macular edema (DME), diabetic retinopathy (DR), and other vascular and inflammatory conditions.

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Key Factors Driving The KLKB1 Inhibitors Market:

• Rising Prevalence of Cardiovascular and Thromboembolic Disorders: Increasing cases of conditions like hereditary angioedema (HAE), thrombosis, and inflammation-related diseases are driving demand for KLKB1 inhibitors.
• Advancements in Targeted Therapies: Growing research in plasma kallikrein biology and drug discovery has enhanced the development of selective KLKB1 inhibitors with improved safety and efficacy profiles.
• Expanding Pipeline of Novel Candidates: Pharmaceutical companies are actively investing in clinical trials for KLKB1 inhibitors, broadening therapeutic applications beyond HAE to cardiovascular and inflammatory disorders.
• Growing Focus on Rare Disease Treatment: Rising awareness and funding for orphan diseases are supporting the clinical and commercial development of KLKB1-targeted drugs.
• Strategic Collaborations and R&D Investments: Partnerships between biotech firms and research institutions are accelerating innovation and regulatory progress in the KLKB1 inhibitors market.

DelveInsight’s “KLKB1 Inhibitors Target Population, Competitive Landscape, and Market Forecast – 2034” report delivers an in-depth understanding of the KLKB1 inhibitors, historical and competitive landscape, as well as the KLKB1 inhibitors therapeutics market trends in the United States, EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan.

The KLKB1 inhibitors market report provides current treatment practices, emerging drugs, market share of individual therapies, and current and forecasted 7MM KLKB1 inhibitors market size from 2020 to 2034. The report also covers current KLKB1 inhibitor treatment practices/algorithms and unmet medical needs to curate the best opportunities and assess the market’s potential.

KLKB1 Inhibitors Disease Understanding

KLKB1 Inhibitors Overview

KLKB1 inhibitors target plasma kallikrein, a central enzyme in the kallikrein–kinin system (KKS) responsible for cleaving high-molecular-weight kininogen into bradykinin, a potent mediator of vasodilation, vascular permeability, pain, and inflammation. Dysregulation of this pathway has been implicated in inflammatory, cardiovascular, metabolic, and thrombotic disorders, making KLKB1 a key node in vascular biology and immune response regulation. Beyond simply blocking bradykinin production, KLKB1 inhibition also influences coagulation cascades and complement system interactions, suggesting a broader role in maintaining vascular homeostasis and modulating pathological inflammation. Recent studies highlight its relevance in retinal vascular leakage, diabetic complications, and even thrombosis risk reduction, expanding its potential significance in disease mechanisms beyond hereditary angioedema.

Further details related to country-based variations are provided in the report...

KLKB1 Inhibitors Epidemiology

The KLKB1 Inhibitors epidemiology chapter in the report provides historical as well as forecasted epidemiology segmented as total cases of selected indications for KLKB1 Inhibitors, total eligible patient pool of selected indications, total treated cases in selected indications for KLKB1 Inhibitors in the 7MM covering the United States, EU4 (Germany, France, Italy, and Spain), and the United Kingdom, and Japan from 2020 to 2034.

Key Findings from Epidemiology Analysis

• As per DelveInsight’s assessment, the total diagnosed prevalent cases of HAE in the 7MM in 2024 were ~14,000, which is expected to increase by 2034, at a considerable CAGR.
• A family history of angioedema helps in the diagnosis, but 25% of cases are new spontaneous mutations. Genetic testing is usually not essential since the biologic tests are relatively accurate after the age of 1 year.
• A higher percentage of diagnosed prevalent cases was observed in the age group 17–65 years in the US, i.e., ~5,000 in 2024, which are anticipated to rise during the forecast period (2025-2034).
• Around 34.2 million individuals in the US are living with diabetes, and nearly 750,000 Americans are affected by DME.
• In the US, it was found that about 10.2% of 22,896 diabetic patients had diabetic retinopathy, and 6.81% had Diabetic Macular Edema.

KLKB1 Inhibitors Drug Chapters

The drug chapter of the KLKB1 report provides an in-depth analysis of pipeline therapies across all stages (Phase I–III), covering clinical trial data, pharmacological profiles, collaborations and agreements, approvals and patents, as well as key advantages, limitations, and recent updates, including news and press releases.

KLKB1 Inhibitors Marketed Drugs

DAWNZERA (donidalorsen): Ionis Pharmaceuticals

DAWNZERA is the first and only RNA-targeted medicine approved for HAE, designed to target plasma prekallikrein (PKK), a key protein that activates inflammatory mediators associated with acute attacks of HAE.  In August 2025, Ionis Pharmaceuticals announced that the US FDA approved DAWNZERA for prophylaxis to prevent attacks of HAE in adult and pediatric patients 12 years of age and older.

EKTERLY (sebetralstat): KalVista Pharmaceuticals

EKTERLY is the first and only oral on-demand treatment for HAE, offering efficacious and safe treatment of attacks without the burden of injections. With ongoing studies exploring its use in children aged two to 11 and multiple regulatory applications under review in key global markets, EKTERLY has the potential to become the foundational therapy for HAE management worldwide.

• In September 2025, KalVista Pharmaceuticals announced that the European Commission and Swissmedic approved EKTERLY for the symptomatic treatment of acute attacks of HAE in adults and adolescents aged 12 years and older.
• In July 2025, KalVista Pharmaceuticals announced that the Medicines and Healthcare products Regulatory Agency (MHRA) of the UK granted marketing authorization, and the US FDA has approved EKTERLY for the treatment of HAE attacks in adults and adolescents aged 12 years and older.

KLKB1 Inhibitors Emerging Drugs

Lonvoguran ziclumeran (lonvo-z): Intellia Therapeutics

Lonvo-z, formerly known as NTLA-2002. Lonvo-z is an investigational in vivo CRISPR-based gene editing therapy designed to prevent HAE attacks by inactivating the KLKB1 gene, which encodes for prekallikrein, the kallikrein precursor protein. Lonvo-z has received five notable regulatory designations, including Orphan Drug and RMAT Designation by the US FDA, the Innovation Passport by the UK MHRA, Priority Medicines (PRIME) Designation by the EMA, as well as Orphan Drug Designation (ODD) by the European Commission.

• In September 2025, Intellia Therapeutics announced it had completed enrollment in the global Phase III HAELO study of lonvoguran ziclumeran for the treatment of hereditary angioedema. Topline data are expected in the first half of 2026. Intellia is on track to submit a Biologics License Application (BLA) in the second half of 2026 to support the company’s plans for a US launch in the first half of 2027.
• In June 2025, Intellia Therapeutics announced three-year follow-up data from the Phase I portion of the ongoing Phase I/II study in patients with HAE after receiving a single dose of lonvo-z (NTLA-2002). Results were shared in an oral presentation at the European Academy of Allergy and Clinical Immunology (EAACI) Congress 2025.

nibart (STAR-0215): Astria Therapeutics

nibart is a monoclonal antibody inhibitor of plasma kallikrein in development for the treatment of HAE. Our goal with navenibart is to provide rapid and sustained HAE attack prevention with a validated mechanism and trusted modality administered subcutaneously every 3 and 6 months. We aim to empower people living with HAE to live life without limitations from their disease.

• In August 2025, Astria Therapeutics announced that it had exclusively licensed development and commercialization rights in Japan to Kaken Pharmaceutical for navenibart, which is in Phase III development for the preventative treatment of HAE.
• In June 2025, Astria Therapeutics announced positive initial results from the target enrollment group in the ongoing ALPHA-SOLAR long-term open-label trial evaluating navenibart in HAE patients. These results demonstrated robust overall reduction in the monthly attack rate (92% mean and 97% median) and further supported its favorable safety and tolerability profile, and potential every three-month- (Q3M) and every six-month (Q6M) dosing regimens.

Note: A Detailed emerging therapies assessment will be provided in the final report...

KLKB1 Inhibitors Market Outlook

The market outlook for KLKB1 inhibitors is strengthening, fueled by increasing recognition of the kallikrein–kinin pathway as a critical mediator in inflammatory, cardiovascular, and rare disease biology. Dysregulation of this pathway underlies conditions such as hereditary angioedema, diabetic complications, and retinal vascular disorders, positioning KLKB1 inhibition as a key therapeutic strategy for diverse indications.

The approvals, including TAKHZYRO (lanadelumab), EKTERLY (sebetralstat), KALBITOR (ecallantide), ORLADEYO (berotralstat), and DAWNZTERA (donidalorsen), along with innovative platforms like NTLA-2002 (CRISPR-based gene editing) and emerging candidates like RZ402 for diabetic macular edema, highlight both the scientific validation and commercial momentum in this space.

Despite these advances, the pipeline remains moderately concentrated, with most assets targeting HAE as the lead indication. This creates opportunities for indication diversification, particularly in diabetic macular edema, cardiovascular inflammation, and thrombotic disorders, where kallikrein dysregulation plays a role.

Overall, KLKB1 inhibitors represent an emerging therapeutic class with the potential to transform multiple disease landscapes. With a favorable mechanism of action, increasing clinical validation, and a growing competitive pipeline, this space is poised for substantial long-term market expansion as innovation moves beyond rare diseases into larger chronic conditions.

KLKB1 Inhibitor Drugs Uptake

This section focuses on the uptake rate of potential emerging KLKB1 inhibitors expected to be launched in the market during 2025–2034.

KLKB1 Inhibitors Pipeline Development Activities

The KLKB1 Inhibitors pipeline report provides insights into different therapeutic candidates in the early stages. It also analyzes key players involved in developing targeted therapeutics.

The presence of numerous drugs under different stages is expected to generate immense opportunities for the KLKB1 inhibitors market growth over the forecasted period.

KLKB1 Inhibitors Pipeline Development Activities

The KLKB1 Inhibitors clinical trials analysis report covers information on collaborations, acquisitions and mergers, licensing, and patent details for KLKB1 inhibitor therapies.

KOL Views on the KLKB1 Inhibitors Market Report

To keep up with current and future market trends, we take Industry Experts’ opinions working in the domain through primary research to fill the data gaps and validate our secondary research. Industry experts were contacted for insights on KLKB1 inhibitors’ evolving treatment landscape, patient reliance on conventional therapies, patient therapy switching acceptability, drug uptake, along challenges related to accessibility.

DelveInsight’s analysts connected with 10+ KOLs to gather insights; however, interviews were conducted with 5+ KOLs in the 7MM. Centers such as Johns Hopkins Sidney Kimmel Cancer Center and others.

Their opinion helps understand and validate current and emerging therapy treatment patterns or KLKB1 inhibitors market trends. This will support the clients in potential upcoming novel treatments by identifying the overall scenario of the market and the unmet needs.

KLKB1 Inhibitors Report Qualitative Analysis

We perform Qualitative and market Intelligence analysis using various approaches, such as SWOT and conjoint analysis. In the SWOT analysis, strengths, weaknesses, opportunities, and threats in terms of gaps in disease diagnosis, patient awareness, physician acceptability, competitive landscape, cost-effectiveness, and geographical accessibility of therapies are provided.

Conjoint Analysis analyzes multiple approved and emerging therapies based on relevant attributes such as safety, efficacy, frequency of administration, route of administration, and order of entry. Scoring is given based on these parameters to analyze the effectiveness of therapy. In efficacy, the trial’s primary and secondary outcome measures are evaluated; for instance, in event-free survival, one of the most important primary outcome measures is event-free survival and overall survival.

Further, the therapies’ safety is evaluated, wherein the acceptability, tolerability, and adverse events are majorly observed, and it sets a clear understanding of the side effects posed by the drug in the trials. In addition, the scoring is also based on the probability of success and the addressable patient pool for each therapy. According to these parameters, the final weightage score and the ranking of the emerging therapies are decided.

KLKB1 Inhibitors Market Access and Reimbursement

Reimbursement may be referred to as the negotiation of a price between a manufacturer and a payer that allows the manufacturer access to the market. It is provided to reduce the high costs and make the essential drugs affordable. Health technology assessment (HTA) plays an important role in reimbursement decision-making and recommending the use of a drug. These recommendations vary widely throughout the seven major markets, even for the same drug.

In the US healthcare system, both Public and Private health insurance coverage are included. Also, Medicare and Medicaid are the largest government-funded programs in the US. The major healthcare programs, including Medicare, Medicaid, the Children's Health Insurance Program (CHIP), and the state and federal health insurance marketplaces, are overseen by the Centers for Medicare & Medicaid Services (CMS). Other than these, Pharmacy Benefit Managers (PBMs) and third-party organizations that provide services and educational programs to aid patients are also present.

The report further provides detailed insights on the country-wise accessibility and reimbursement scenarios, cost-effectiveness scenario of approved therapies, programs making accessibility easier and out-of-pocket costs more affordable, insights on patients insured under federal or state government prescription drug programs, etc.

Scope of the KLKB1 Inhibitors Market Report

• The report covers a segment of key events, an executive summary, and a descriptive overview of the KLKB1 inhibitor, explaining its mechanism and emerging therapies.
• Comprehensive insight into the competitive landscape, and forecasts, the future growth potential of treatment rate, drug uptake, and drug information has been provided.
• Additionally, an all-inclusive account of the current and emerging therapies and the elaborate profiles of late-stage and prominent therapies will impact the current landscape.
• A detailed review of the KLKB1 inhibitor market, historical and forecasted market size, market share by therapies, detailed assumptions, and rationale behind our approach is included in the report, covering the 7MM drug outreach.
• The report provides an edge while developing business strategies, by understanding trends, through SWOT and conjoint analysis, expert insights/KOL views, and treatment preferences that help shape and drive the 7MM KLKB1 inhibitor market.

KLKB1 Inhibitors Market Report Insights

• KLKB1 Inhibitor Targeted Patient Pool
• KLKB1 Inhibitors Therapeutic Approaches
• KLKB1 Inhibitor Pipeline Analysis
• KLKB1 Inhibitor Market Size and Trends
• Existing and future Market Opportunity

KLKB1 Inhibitor Market Report Key Strengths

• Ten-Year Forecast
• The 7MM Coverage
• Key Cross Competition
• KLKB1 Inhibitors Drugs Uptake
• Key KLKB1 Inhibitors Market Forecast Assumptions

KLKB1 Inhibitor Market Report Assessment

• Current KLKB1 Inhibitors Treatment Practices
• KLKB1 Inhibitors Unmet Needs
• KLKB1 Inhibitors Analyst Views
• KLKB1 Inhibitors Pipeline Product Profiles
• KLKB1 Inhibitors Market Attractiveness
• Qualitative Analysis (SWOT and Conjoint Analysis)
• KLKB1 Inhibitors Market Drivers
• KLKB1 Inhibitors Market Barriers

Key Questions Questions Answered In The KLKB1 Inhibitors Market Report:

• What was the KLKB1 inhibitor total market size, the market size by therapies, market share (%) distribution, and what would it look like in 2034? What are the contributing factors for this growth?
• Which drug is going to be the largest contributor in 2034?
• What are the risks, burdens, and unmet needs of treatment with KLKB1 inhibitors? What will be the growth opportunities across the 7MM for the patient population of KLKB1 inhibitors?
• What are the key factors hampering the growth of the KLKB1 inhibitor market?
• What are the indications for which recent novel therapies and technologies have been developed to overcome the limitations of existing treatments?
• What key designations have been granted to the therapies for KLKB1 inhibitors?
• Patient acceptability in terms of preferred therapy options as per real-world scenarios?

Reasons to buy KLKB1 Inhibitors Market Forecast Report

• The report will help develop business strategies by understanding the latest trends and changing dynamics driving the KLKB1 inhibitor Market.
• Understand the existing market opportunities in varying geographies and the growth potential over the coming years.
• Distribution of historical and current patient share based on real-world prescription data along with reported sales of approved products in the US, EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan.
• Identifying strong upcoming players in the market will help devise strategies to help get ahead of competitors.
• To understand Key Opinion Leaders’ perspectives around the accessibility, acceptability, and compliance-related challenges of existing treatment to overcome barriers in the future.
• Detailed insights into the unmet needs of the existing market so that the upcoming players can strengthen their development and launch strategy.