세계의 판코니 빈혈 치료제 시장 : 치료 유형별, 연령층별, 병기별, 지역별

Fanconi Anemia Drug Market is estimated to be valued at USD 634.1 Mn in 2025 and is expected to reach USD 898.2 Mn by 2032, growing at a compound annual growth rate (CAGR) of 5.1% from 2025 to 2032.

The fanconi anemia drugs are pharmaceutical therapies and treatments that have been created or are currently being researched with the goal of treating the symptoms and underlying genetic abnormalities of fanconi anemia, a rare genetic disorder that is marked by bone marrow failure, congenital defects, and an increased risk of developing cancer. Numerous organ systems are impacted by fanconi anemia. Fanconi anemia results from a defective gene that harms cells, and prevents them from mending damaged DNA. It has an impact on practically all body organs. It is also known as aplastic anemia that is hereditary.

Market Dynamics:

Continuous advancements in scientific research and knowledge about molecular mechanisms are expected to drive the global fanconi anemia drug market growth over the forecast period. Substantial unmet medical need will also continue to fuel growth of the global fanconi anemia drug market over the forecast period. This element motivates efforts in research and development to find efficient therapeutics for the control and treatment of FA. Development of disease-modifying therapies will create new opportunities for the global fanconi anemia drug market over the forecast period. There are opportunities for the creation of disease-modifying medications that can deal with the genetic alterations and molecular mechanisms connected to FA. For instance, on May 17, 2022, Jasper Therapeutics, Inc., a biotechnology company, announced the dosing of the first patient in its sponsored research of the Center for Definitive and Curative Medicine (CDCM) at the Stanford University School of Medicine for the study of JPS191 as a conditioning agent in the treatment of Fanconi Anemia. In accordance with the sponsored research agreement, Stanford Medicine is conducting a Phase 1/2 clinical trial (NCT04784052) utilizing JSP191 to treat Fanconi Anemia patients in bone marrow failure requiring allogeneic transplant with non-sibling donors.

Key features of the study:

• This report provides in-depth analysis of the global fanconi anemia drug market, and provides market size (US$ Mn) and compound annual growth rate (CAGR%) for the forecast period (2025-2032), considering 2024 as the base year
• It elucidates potential revenue opportunities across different segments and explains attractive investment proposition matrices for this market
• This study also provides key insights about market drivers, restraints, opportunities, new product launches or approval, market trends, regional outlook, and competitive strategies adopted by key players
• It profiles key players in the global fanconi anemia drug market based on the following parameters - company highlights, products portfolio, key highlights, financial performance, and strategies
• Key companies covered as a part of this study include Foresee Pharmaceuticals Co., Ltd., Rocket Pharmaceuticals Inc., Amgen, Novartis AG, Gilead Sciences, Inc. (Kite Pharma, Inc.), Celgene Corporation (Juno Therapeutics), Spark Therapeutics, UniQure N.V, Bluebird Bio, Inc., PTC Therapeutics, Orchard Therapeutics Limited, GlaxoSmithKline Plc., Aurora Biopharma, Inc., Pfizer, Inc. and Autolus Therapeutics
• Insights from this report would allow marketers and the management authorities of the companies to make informed decisions regarding their future product launches, type up-gradation, market expansion, and marketing tactics
• Global fanconi anemia drug market report caters to various stakeholders in this industry including investors, suppliers, product manufacturers, distributors, new entrants, and financial analysts
• Stakeholders would have ease in decision-making through various strategy matrices used in analyzing the global fanconi anemia drug market

Detailed Segmentation:

• By Treatment Type:
  • Hematopoietic Growth Factors
  • Gene Therapy
  • Stem Cell Transplantation
  • Other Therapies
• By Age Group:
  • Pediatric Patients
  • Adult Patients
• By Disease Stage:
  • Early-stage Disease
  • Advanced-stage Disease
• By Region:
  • North America
  • Europe
  • Asia Pacific
  • Latin America
  • Middle East
  • Africa
• Company Profiles:
  • Foresee Pharmaceuticals Co., Ltd.
  • Rocket Pharmaceuticals Inc.
  • Amgen
  • Novartis AG
  • Gilead Sciences, Inc. (Kite Pharma, Inc.)
  • Celgene Corporation (Juno Therapeutics)
  • Spark Therapeutics
  • UniQure N.V
  • Bluebird Bio, Inc.
  • PTC Therapeutics
  • Orchard Therapeutics Limited
  • GlaxoSmithKline Plc
  • Aurora Biopharma, Inc.
  • Pfizer, Inc.
  • Autolus Therapeutics

Table of Contents

1. Research Objectives and Assumptions

• Research Objectives
• Assumptions
• Abbreviations

2. Market Purview

• Report Description
  • Market Definition and Scope
• Executive Summary
  • Market Fanconi Anemia Drug, By Treatment Type
  • Market Fanconi Anemia Drug, By Age Group
  • Market Fanconi Anemia Drug, By Disease Stage
  • Market Fanconi Anemia Drug, By Region
• Coherent Opportunity Map (COM)

3. Market Dynamics, Regulations, and Trends Analysis

• Market Dynamics
  • Drivers
• Continuous advancements in scientific research and understanding of the molecular mechanisms
• Substantial unmet medical need
  • Restraints
• Lack of recognized therapies
  • Opportunities
• Development of Disease-Modifying Therapies
• Impact Analysis
• Key Highlights
• Regulatory Scenario
• Product Launches/Approvals
• PEST Analysis
• PORTER's Analysis
• Merger and Acquisition Scenario

4. Global Fanconi Anemia Drug Market - Impact of Coronavirus (COVID-19) Pandemic

• COVID-19 Epidemiology
• Supply Side and Demand Side Analysis
• Economic Impact

5. Global Fanconi Anemia Drug Market, By Treatment Type, 2020-2032, (US$ Mn)

• Introduction
  • Market Share Analysis, 2025 and 2032 (%)
  • Y-o-Y Growth Analysis, 2021-2032
  • Segment Trends
• Hematopoietic Growth Factors
  • Introduction
  • Market Size and Forecast, and Y-o-Y Growth, 2020-2032, (US$ Mn)
• Gene Therapy
  • Introduction
  • Market Size and Forecast, and Y-o-Y Growth, 2020-2032, (US$ Mn)
• Stem Cell Transplantation
  • Introduction
  • Market Size and Forecast, and Y-o-Y Growth, 2020-2032, (US$ Mn)
• Other Therapies
  • Introduction
  • Market Size and Forecast, and Y-o-Y Growth, 2020-2032, (US$ Mn)

6. Global Fanconi Anemia Drug Market, By Age Group, 2020-2032, (US$ Mn)

• Introduction
  • Market Share Analysis, 2025 and 2032 (%)
  • Y-o-Y Growth Analysis, 2021-2032
  • Segment Trends
• Pediatric Patients
  • Introduction
  • Market Size and Forecast, and Y-o-Y Growth, 2020-2032, (US$ Mn)
• Adult Patients
  • Introduction
  • Market Size and Forecast, and Y-o-Y Growth, 2020-2032, (US$ Mn)

7. Global Fanconi Anemia Drug Market, By Disease Stage, 2020-2032, (US$ Mn)

• Introduction
  • Market Share Analysis, 2025 and 2032 (%)
  • Y-o-Y Growth Analysis, 2021-2032
  • Segment Trends
• Early-stage Disease
  • Introduction
  • Market Size and Forecast, and Y-o-Y Growth, 2020-2032, (US$ Mn)
• Advanced-stage Disease
  • Introduction
  • Market Size and Forecast, and Y-o-Y Growth, 2020-2032, (US$ Mn)

8. Global Fanconi Anemia Drug Market, By Region, 2020-2032, (US$ Mn)

• Introduction
  • Market Share Analysis, By Region, 2025 and 2032 (%)
  • Y-o-Y Growth Analysis, For Region, 2021 -2032
  • Country Trends
• North America
  • Introduction
  • Market Size and Forecast, and Y-o-Y Growth, By Treatment Type, 2020-2032, (US$ Mn)
  • Market Size and Forecast, and Y-o-Y Growth, By Age Group, 2020-2032, (US$ Mn)
  • Market Size and Forecast, and Y-o-Y Growth, By Disease Stage, 2020-2032, (US$ Mn)
  • Market Size and Forecast, and Y-o-Y Growth, By Country, 2020-2032,(US$ Mn)
    • U.S.
    • Canada
• Latin America
  • Introduction
  • Market Size and Forecast, and Y-o-Y Growth, By Treatment Type, 2020-2032, (US$ Mn)
  • Market Size and Forecast, and Y-o-Y Growth, By Age Group, 2020-2032, (US$ Mn)
  • Market Size and Forecast, and Y-o-Y Growth, By Disease Stage, 2020-2032, (US$ Mn)
  • Market Size and Forecast, and Y-o-Y Growth, By Country, 2020-2032,(US$ Mn)
    • Brazil
    • Argentina
    • Mexico
    • Rest of Latin America
• Europe
  • Introduction
  • Market Size and Forecast, and Y-o-Y Growth, By Treatment Type, 2020-2032, (US$ Mn)
  • Market Size and Forecast, and Y-o-Y Growth, By Age Group, 2020-2032, (US$ Mn)
  • Market Size and Forecast, and Y-o-Y Growth, By Disease Stage, 2020-2032, (US$ Mn)
  • Market Size and Forecast, and Y-o-Y Growth, By Country, 2020-2032,(US$ Mn)
    • Germany
    • U.K.
    • Spain
    • France
    • Italy
    • Russia
    • Rest of Europe
• Asia Pacific
  • Introduction
  • Market Size and Forecast, and Y-o-Y Growth, By Treatment Type, 2020-2032, (US$ Mn)
  • Market Size and Forecast, and Y-o-Y Growth, By Age Group, 2020-2032, (US$ Mn)
  • Market Size and Forecast, and Y-o-Y Growth, By Disease Stage, 2020-2032, (US$ Mn)
  • Market Size and Forecast, and Y-o-Y Growth, By Country, 2020-2032,(US$ Mn)
    • China
    • India
    • Japan
    • Australia
    • South Korea
    • ASEAN
    • Rest of Asia Pacific
• Middle East
  • Introduction
  • Market Size and Forecast, and Y-o-Y Growth, By Treatment Type, 2020-2032, (US$ Mn)
  • Market Size and Forecast, and Y-o-Y Growth, By Age Group, 2020-2032, (US$ Mn)
  • Market Size and Forecast, and Y-o-Y Growth, By Disease Stage, 2020-2032, (US$ Mn)
  • Market Size and Forecast, and Y-o-Y Growth, By Country, 2020-2032,(US$ Mn)
    • GCC
    • Israel
    • Rest of Middle East
• Africa
  • Introduction
  • Market Size and Forecast, and Y-o-Y Growth, By Treatment Type, 2020-2032, (US$ Mn)
  • Market Size and Forecast, and Y-o-Y Growth, By Age Group, 2020-2032, (US$ Mn)
  • Market Size and Forecast, and Y-o-Y Growth, By Disease Stage, 2020-2032, (US$ Mn)
  • Market Size and Forecast, and Y-o-Y Growth, By Country, 2020-2032,(US$ Mn)
    • South Africa
    • North Africa
    • Central Africa

9. Competitive Landscape

• Foresee Pharmaceuticals Co., Ltd.
  • Company Highlights
  • Product Portfolio
  • Key Developments
  • Financial Performance
  • Strategies
• Rocket Pharmaceuticals Inc.
  • Company Highlights
  • Product Portfolio
  • Key Highlights
  • Financial Performance
  • Strategies
• Amgen
  • Company Highlights
  • Product Portfolio
  • Key Highlights
  • Financial Performance
  • Strategies
• Novartis AG
  • Company Highlights
  • Product Portfolio
  • Key Highlights
  • Financial Performance
  • Strategies
• Gilead Sciences, Inc. (Kite Pharma, Inc.)
  • Company Highlights
  • Product Portfolio
  • Key Highlights
  • Financial Performance
  • Strategies
• Celgene Corporation (Juno Therapeutics)
  • Company Highlights
  • Product Portfolio
  • Key Highlights
  • Financial Performance
  • Strategies
• Spark Therapeutics
  • Company Highlights
  • Product Portfolio
  • Key Highlights
  • Financial Performance
  • Strategies
• UniQure N.V
  • Company Highlights
  • Product Portfolio
  • Key Highlights
  • Financial Performance
  • Strategies
• Bluebird Bio, Inc.
  • Company Highlights
  • Product Portfolio
  • Key Highlights
  • Financial Performance
  • Strategies
• PTC Therapeutics
  • Company Highlights
  • Product Portfolio
  • Key Highlights
  • Financial Performance
  • Strategies
• Orchard Therapeutics Limited
  • Company Highlights
  • Product Portfolio
  • Key Highlights
  • Financial Performance
  • Strategies
• GlaxoSmithKline Plc.
  • Company Highlights
  • Product Portfolio
  • Key Highlights
  • Financial Performance
  • Strategies
• Aurora Biopharma, Inc.
  • Company Highlights
  • Product Portfolio
  • Key Highlights
  • Financial Performance
  • Strategies
• Pfizer, Inc.
  • Company Highlights
  • Product Portfolio
  • Key Highlights
  • Financial Performance
  • Strategies
• Autolus Therapeutics
  • Company Highlights
  • Product Portfolio
  • Key Highlights
  • Financial Performance
  • Strategies
• Analyst Views

10. Section

• Research Methodology
• About us