혈우병 A 및 B용 세포·유전자 치료(CGT)

Clients will gain insight into the competitive landscape of leading CGT agents in Hemophilia A & B including launch date projections, key clinical trial analysis, analyst consensus forecasts, likelihood of approval analysis, and commentary on current and future players. The report also includes outlook from four key opinion leaders in the 5EU, US, and China and analyzes key unmet needs in the space. Additionally, the report includes commentary on the regulatory landscape of CGTs and on the reimbursement environment.

The global hemophilia A and B CGT market is expected to see one of the most massive expansions across therapeutic areas, climbing from $12 million in 2023 to more than $2.2 billion by 2029.

Disease Overview

GlobalData estimates there are 45,761 and 9,335 patients with hemophilia A or B, without factor inhibitor, in the 8MM in 2024.

Current Treatment Options

Current adeno-associated virus (AAV)-based GTs, Roctavian and Hemgenix, aim to provide long-term bleed protection, minimizing burden on patients.

CSL Behring is more robust in HCP engagement than BioMarin, thanks to its more comprehensive, omni-channel reach to both patients and hematologists.

Current and pipeline hemophilia CGTs are not robust enough to address the concerns on the modality's durability, safety and patient access.

Future Market Assessment

Hemophilia A and B have a relatively lean CGT pipeline with 14 GTs and 1 gene-modified cell therapy currently in clinical trials. Most of these are in Phase I/II.

Within two years, the hemophilia space will see greater competition pressure due to the entry of well-established pharmaceuticals with strong marketing capability.

Long-term efficacy evidence and comparison over modern standard-of-care are top concerns from agencies on evaluating hemophilia GTs.

The limited geographical reach of GTs from established pharmaceuticals provides a market opportunity for biotechs with niche modality focus and domestic expertise to emerge.

Key Highlights

Clinical trial analysis includes all countries

4 KOLs interviewed

Scope

Contents include -

Executive Summary

Disease Overview

Current Treatment Options and Unmet Needs

Future Market Assessment

Likelihood of Approval and Phase Transition Success Rate Analysis

Appendix

Reasons to Buy

OBTAIN A COMPETITIVE ASSESMENT FOR CELL & GENE THERAPIES IN HEMOPHILIA A & B ACROSS MULTIPLE REGIONS

Our modality-specific reports answer questions such as -

• What is the future global market value of CGT in Hemophilia A&B?
• Which patient groups are more likely to receive these therapies?
• What is the price of these agents and what is the reimbursement landscape?
• What is the key opinion leaders' outlook for the products?
• What are key launch dates and other catalysts in the area?

Table of Contents

Table of Contents

1. Preface

• 1.1. Contents
• 1.2. Abbreviations
• 1.3. Related Reports

2. Executive Summary

3. Disease Overview

• 3.1. Overview of Hemophilia A & B

4. Current Treatment Options

• 4.1. Hemophilia GTs Provide Unmatched Long-Term Bleed Protection Despite a More Stringent Patient Requirement
• 4.2. KOLs Highlighted Vastly Different Priorities From Physician and Patient Perspectives on GTs
• 4.3. BioMarin and CSL Behring Have Adopted Similar Messaging and Marketing Strategies
• 4.4. Medical Needs in Hemophilia A & B Are Not Adequately Addressed in the Near Future
• 4.5. KOLs Shared their Concerns on the Limitations of Current Hemophilia Gene Therapies

5. Future Market Assessment

• 5.1. Major Players Are Expected To Intensify the Competition Within a Year's Time
• 5.2. Reimbursement Uncertainties Exist for Hemophilia Gene Therapies
• 5.3. The FDA Provides Industry Guidance on Developing Hemophilia Gene Therapies
• 5.4. KOLs Highlighted the Difficulties With Reimbursement for GTs While PROs May Add Support
• 5.5. Hemophilia A & B Pipeline Is Concentrated in Phase I/II Trials
• 5.6. Hemophilia A CGT Landscape Is Becoming More Active With Diversified Players
• 5.7. Early Hemophilia B GT Landscape Is Concentrated With Developers From Asia
• 5.8. KOLs Compares the Narrower Patient Eligibility of Pfizer's Beqvez With CSL's Hemgenix
• 5.9. KOLs Have Reservations on SPK-8011 and the ET-3 Payload-Carrying GTs
• 5.10. Hemophilia CGT Segment Are Expected to Pass $2.14 Billion by 2029
• 5.11. Hemophilia CGT Landscape Is Mixed With a Wide Variety of Developers

6. Likelihood of Approval and Phase-Transition Success Rate Analysis

• 6.1. Hemophilia CGT Candidates Have Varying LOAs and PTSRs

7. Appendix

• 7.1. Primary Research: Key Opinion Leader Information
• 7.2. Bibliography
• 7.3. About the Authors

8. Contact Us