C5 보체 억제제 시장 - 표적 집단, 경쟁 구도, 시장 예측(2034년)

Key Highlights:

• C5 inhibitor exerts its effect by targeting the complement protein C5 and preventing it from splitting into C5a and C5b fragments, which play a major role in the formation of membrane attack complex (MAC). This averts the complement-mediated lysis of RBCs (Intravascular hemolysis), however it fails to stop the extravascular hemolysis.
• Currently, there are five C5 complement inhibitors in the market, approved for various complement dysregulation disorders.
• Alexion's SOLIRIS (eculizumab) was the first complement inhibitor approved in 2007 for Paroxysmal Nocturnal Hemoglobinuria (PNH). It was later approved for additional three indications namely, Atypical Hemolytic Uremic Syndrome (aHUS), Neuromyelitis Optica Spectrum Disorder (NMOSD), and Generalized Myasthenia Gravis (gMG).
• In addition to SOLIRIS and ULTOMIRIS, the market for C5 complement inhibitors includes Astellas' IZERVAY (avacincaptad pegol), which is approved for geographic atrophy, Regeneron's VEOPOZ (Pozelimab), approved for Chaple disease, and UCB Pharma's ZILBRYSQ (zilucoplan), approved for generalized myasthenia gravis.
• A few of the potential emerging C5 complement inhibitors are crovalimab, nomacopan (C5/LTB4 inhibitor), CAN-106 (C5 inhibitor), and KP104 (C5 and Factor H).
• In July 2022, FDA granted Orphan Drug Designation (ODD) to KP104 for the treatment of PNH.
• The current market size of C5 complement inhibtors is estimated to be approximately USD 4 Billion, in the 7 MM.

DelveInsight's "C5 Complement inhibitor - Target Population, Competitive Landscape, and Market Forecast - 2034" report delivers an in-depth understanding of the C5 complement inhibitors, historical and forecasted epidemiology, competitive landscape as well as the C5 complement inhibitors market trends in the United States, EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan.

The C5 complement inhibitors market report provides current treatment practices, emerging drugs, market share of individual therapies, and current and forecasted 7MM C5 complement inhibitors market size from 2020 to 2034. The report also covers current C5 complement inhibitors treatment practices/algorithms and unmet medical needs to curate the best opportunities and assess the market's potential.

Geography Covered:

• The United States
• EU4 (Germany, France, Italy, and Spain) and the United Kingdom
• Japan

Study Period: 2020-2034

C5 Complement Inhibitors Disease Understanding and Treatment Algorithm

C5 Complement Inhibitors Overview

C5 is the initiator of the effector terminal phase of the complement system and shares the molecular structure consisting of two chains-a and B, linked by disulfide bonds, with C3 and C4, with the only difference being that C5 does not contain an internal thio-ester bond. The terminal phase is similar for the classical, lectin, and alternative pathways. The incorporation of C3b in the C3 convertases results in the formation of the C5 convertases: C3bBbC3b for the AP and C4bC2aC3b for the CP and LP. These C5 convertases cleave C5 into C5a and C5b, ultimately resulting in the formation of the multimeric MAC.

C5 Complement Inhibitors Treatment

SOLIRIS (eculizumab) and ULTOMIRIS (ravulizumab) are some of the few terminal complement inhibitors that block the activity of C5, preventing the formation of the C5b-9 terminal complement complex. These agents inhibit intravascular hemolysis mediated by the terminal complement in patients with complement dysregulation disorders. Despite demonstrated benefits in various complement-mediated diseases, use of inhibitors targeting the classical/lectin and terminal pathways is associated with limitations. Some patients with PNH remain anemic and still require transfusions despite treatment with a C5 or C3 inhibitor.

C5 Complement Inhibitors Drug Chapters

The drug chapter segment of the complement inhibitors reports encloses a detailed analysis of complement inhibitors' marketed drugs and late-stage (Phase III and Phase II) pipeline drugs. It also helps understand the C5 complement inhibitors' clinical trial details, expressive pharmacological action, agreements and collaborations, approval and patent details, advantages and disadvantages of each included drug and the latest news and press releases.

Marketed Drugs

Currently, there are five marketed C5 complement inhibitors in various indications.

ZILBRYSQ (zilucoplan) - UCB Pharma

Zilucoplan is a once-daily, subcutaneous, self-administered peptide inhibitor of complement component 5 (C5 inhibitor). As the only once-daily gMG target therapy for self-administration by adult patients with anti-AChR antibody-positive gMG, zilucoplan inhibits complement-mediated damage to the neuromuscular junction through its targeted mechanism of action. The drug received FDA approval in October 2023.

Moroever, in September 2023, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) issued a positive opinion recommending granting marketing authorization for zilucoplan in the European Union (EU) as an add-on to standard therapy for the treatment of generalized myasthenia gravis (gMG) and was later granted marketing authorization by EMA in December 2023. Also in the same year, Japanese Ministry of Health, Labour and Welfare (MHLW) approved zilucoplan for the treatment of gMG in adult patients who inadequately respond to steroids or other immunosuppressants.

Emerging Drugs

Crovalimab: Genentech

Crovalimab is an investigational, novel anti-C5 recycling monoclonal antibody designed to block the complement system. Crovalimab works by binding to C5, blocking the last step of the complement cascade and is also recycled within the bloodstream, enabling rapid and sustained complement inhibition. Crovalimab's recycling properties also enable low dose subcutaneous (SC) administration every four weeks. In addition, crovalimab binds to a different C5 binding site from current treatments, which has the potential to provide a treatment option for people with specific C5 gene mutations who do not respond to current therapies. It is also being evaluated in atypical hemolytic uremic syndrome, sickle cell disease, and other complement mediated diseases

In September 2023, FDA accepted the Biologics License Application (BLA) for crovalimab, for the treatment of paroxysmal nocturnal hemoglobinuria (PNH). If approved, crovalimab will be the first monthly subcutaneous treatment for PNH, with the option to self-administer outside of a supervised healthcare setting

C5 Complement Inhibitors Market Outlook

The market for C5 complement inhibitors is poised for substantial growth in the upcoming years. Following the introduction of SOLIRIS and ULTOMIRIS, there has been considerable momentum in the complement inhibitor market. Numerous companies are currently in the process of developing innovative complement inhibitors with the aim of matching the efficacy and safety standards set by eculizumab.

Since the PNH and aHUS has already been crowded with a lot of marketed and emerging drugs. The companies have now shifted their focus to other complement dysregulation disorders, including but not limited to C3G, IgA-N, and Geographic Atrophy.

Besides major players, small pharma companies are currently assessing innovative C5 inhibitors for a range of complement-mediated disorders, with an aim to offer more convenient routes of administration or target multiple proteins simultaneously. For example, Genentech's crovalimab provides subcutaneous delivery, whereas Akari Therapeutics' nomacopan inhibits both C5 and LTB4.

C5 Complement Inhibitors Drugs Uptake

This section focuses on the uptake rate of potential approved and emerging C5 Complement inhibitors expected to be launched in the market during 2020-2034.

C5 Complement Inhibitors Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase III phase II, and phase I. It also analyzes key players involved in developing targeted therapeutics.

The presence of numerous drugs under different stages is expected to generate immense opportunity for C5 Complement Inhibitors market growth over the forecasted period.

Key Events

• The report covers information on collaborations, acquisitions and mergers, licensing, and patent details for complement inhibitors emerging therapies.
• The increasing strategic collaborations among major market players to enhance the growth of their pipeline products are anticipated to drive market expansion.
• In 2021, AstraZeneca acquired Alexion for USD 39 billion, with an aim to create a dedicated rare disease unit and enter into the market of other complement mediated disorders through Alexions' pipeline.
• In 2022, Amgen acquired ChemoCentryx for USD 3.7 billion, in order to retain its share in the inflammatory disease market with ChemoCentryxs' TAVNEOS (avocapan). Avocapan is approved for ANCA-associated vasculitis and is currently being evaluated for Hidradenitis Suppurativa in a phase II study.
• In 2023, Astellas acquired Iveric Bio for USD 5.9 billion. Iveric Bio's IZERVAY (avacincaptad pegol) is approved for geographic atrophy.

KOL Views

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 C5 complement 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 25+ KOLs to gather insights; however, interviews were conducted with 10+ KOLs in the 7MM.

Their opinion helps understand and validate current and emerging therapy treatment patterns or C5 complement inhibitors market trends. This will support the clients in understanding potential upcoming novel treatments by identifying the overall scenario of the market and the unmet needs.

Qualitative Analysis

We perform Qualitative and market Intelligence analysis using various approaches, such as SWOT analysis. In the SWOT analysis, strengths, weaknesses, opportunities, and threats in terms of disease diagnosis, patient awareness, patient burden, competitive landscape, cost-effectiveness, and geographical accessibility of therapies are provided. These pointers are based on the analyst's discretion and assessment of the patient burden, cost analysis, and existing and evolving treatment landscape.

Scope of the Report:

• The report covers a segment of key events, an executive summary, and a descriptive overview of C5 complement inhibitors, explaining their mechanism, and therapies (current and emerging).
• Comprehensive insight into the competitive landscape, forecasts, the future growth potential of treatment rate, drug uptake, and drug information have been provided.
• Additionally, an all-inclusive account of the current and emerging therapies and the elaborative profiles of late-stage and prominent therapies that will impact the current landscape.
• A detailed review of the C5 complement Inhibitors 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 analysis, expert insights/KOL views, and treatment preferences that help shape and drive the 7MM Complement inhibitors market.

C5 complement inhibitors report insights

• C5 Complement Inhibitors Targeted Patient Pool
• Therapeutic Approaches
• C5 Complement Inhibitors Pipeline Analysis
• C5 Complement Inhibitors Market Size and Trends
• Existing and Future Market Opportunity

C5 complement inhibitors Report Key Strengths

• Eleven years Forecast
• The 7MM Coverage
• Key Cross Competition
• Drugs Uptake and Key Market Forecast Assumptions

C5 complement inhibitors Report Assessment

• Current Treatment Practices
• Unmet Needs
• Pipeline Product Profiles
• Market Attractiveness
• Qualitative Analysis (SWOT)

Key Questions:

• What was the C5 complement inhibitors 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?
• Which is the most lucrative market for C5 complement inhibitors?
• Which drug type segment accounts for maximum C5 complement inhibitors sales?
• What are the pricing variations among different geographies for approved therapies?
• How has the reimbursement landscape for C5 complement inhibitors evolved since the first one was approved? Do patients have any access issues that are driven by reimbursement decisions?
• What are the risks, burdens, and unmet needs of treatment with C5 complement inhibitors? What will be the growth opportunities across the 7MM for the patient population of C5 complement inhibitors?
• What are the key factors hampering the growth of the C5 complement inhibitors 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 C5 complement inhibitors?
• What is the cost burden of approved therapies on the patient?
• Patient acceptability in terms of preferred therapy options as per real-world scenarios?
• What are the country-specific accessibility issues of expensive, recently approved therapies?

Reasons to buy:

• The report will help develop business strategies by understanding the latest trends and changing dynamics driving the C5 complement inhibitors 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) the United Kingdom, and Japan.
• Identifying strong upcoming players in the market will help devise strategies to help get ahead of competitors.
• Detailed analysis and ranking of indication-wise current and emerging therapies under the conjoint analysis section to provide visibility around leading indications.
• To understand Key Opinion Leaders' perspectives around the accessibility, acceptability, and compliance-related challenges to overcome barriers in the future.
• Detailed insights on the unmet needs of the existing market so that the upcoming players can strengthen their development and launch strategy.

Table of Contents

1. Key Insights

2. Report Introduction

3. Executive Summary of C5 Complement Inhibitors

4. Key Events

5. Market Forecast Methodology

6. C5 Complement Inhibitors Market Overview at a Glance in the 7MM

• 6.1. Market Share (%) Distribution by Indication in 2020
• 6.2. Market Share (%) Distribution by Indication in 2034

7. C5 Complement Inhibitors: Background and Overview

• 7.1. Introduction
• 7.2. Evolution of C5 Complement Inhibitors
• 7.3. Treatment

8. Target Patient Pool

• 8.1. Key Findings
• 8.2. Assumptions and Rationale: 7MM
• 8.3. Epidemiology Scenario in the 7MM
  • 8.3.1. Total Prevalent Cases in Selected Indications for C5 Complement Inhibitors in the 7MM
  • 8.3.2. Total Eligible Patient Pool for C5 Complement Inhibitors in Selected Indications in the 7MM
  • 8.3.3. Total Treated Cases in Selected Indications for C5 Complement Inhibitors in the 7MM
• 8.4. Epidemiology Scenario in the United States
  • 8.4.1. Total Prevalent Cases in Selected Indications for C5 Complement Inhibitors in the United States
  • 8.4.2. Total Eligible Patient Pool for C5 Complement Inhibitors in Selected Indications in the United States
  • 8.4.3. Total Treated Cases in Selected Indications for C5 Complement Inhibitors in the United States
• 8.5. Epidemiology Scenario in EU4 and the UK
  • 8.5.1. Total Prevalent Cases in Selected Indications for C5 Complement Inhibitors in EU4 and the UK
  • 8.5.2. Total Eligible Patient Pool for C5 Complement Inhibitors in Selected Indications in EU4 and the UK
  • 8.5.3. Total Treated Cases in Selected Indications for C5 Complement Inhibitors in EU4 and the UK
• 8.6. Epidemiology Scenario in Japan
  • 8.6.1. Total Prevalent Cases in Selected Indications for C5 Complement Inhibitors in Japan
  • 8.6.2. Total Eligible Patient Pool for C5 Complement Inhibitors in Selected Indications in Japan
  • 8.6.3. Total Treated Cases in Selected Indications for C5 Complement Inhibitors in Japan

9. Marketed Therapies

• 9.1. Key Competitors
• 9.2. ZILBRYSQ (zilucoplan) : UCB Pharma
  • 9.2.1. Product Description
  • 9.2.2. Other developmental activities
  • 9.2.3. Clinical development
  • 9.2.4. Safety and efficacy

10. Emerging Therapies

• 10.1. Key Competitors
• 10.2. Crovalimab : Genentech
  • 10.2.1. Product Description
  • 10.2.2. Other developmental activities
  • 10.2.3. Clinical development
  • 10.2.4. Safety and efficacy

11. C5 Complement Inhibitors: Seven Major Market Analysis

• 11.1. Key Findings
• 11.2. Market Outlook
• 11.3. Key Market Forecast Assumptions
• 11.4. Total Market Size of C5 Complement Inhibitors in the 7MM
• 11.5. Market Size of C5 Complement Inhibitors by Indication in the7MM
• 11.6. The United States Market Size
  • 11.6.1. Total Market Size of C5 Complement Inhibitors in the United States
  • 11.6.2. Market Size of C5 Complement Inhibitors by Indication in the United States
  • 11.6.3. Market Size of C5 Complement Inhibitors by Therapies in the United States
• 11.7. EU4 and the UK Market Size
  • 11.7.1. Total Market Size of C5 Complement Inhibitors in EU4 and the UK
  • 11.7.2. Market Size of C5 Complement Inhibitors by Indication in EU4 and the UK
  • 11.7.3. Market Size of C5 Complement Inhibitors by Therapies in EU4 and the UK
• 11.8. Japan Market Size
  • 11.8.1. Total Market Size of C5 Complement Inhibitors in Japan
  • 11.8.2. Market Size of C5 Complement Inhibitors by Indication in Japan
  • 11.8.3. Market Size of C5 Complement Inhibitors by Therapies in Japan

12. Market Access and Reimbursement

13. SWOT Analysis

14. KOL Views

15. Unmet Needs

16. Appendix

• 16.1. Bibliography
• 16.2. Report Methodology

17. DelveInsight Capabilities

18. Disclaimer

19. About DelveInsight