Cell and Gene Therapies in Hemophilia A & B

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

• The publisher 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?