한글목차

CASGEVY의 성장을 이끄는 주요 요인들

1. 시장 점유율 확대 및 신규 환자 수 증가

• CASGEVY는 평생에 걸친 지지요법에 비해 1회 투여로 완치 가능성이 있는 치료 프로파일을 가지고 있으며, 특히 겸상적혈구증(SCD)과 수혈 의존성 베타 지중해빈혈(TDT) 등 희귀혈액질환 시장에서 빠르게 채택되고 있습니다.
• 적응증 확대와 치료 시설 증가가 환자 수 증가를 견인하고 있으며, 전 세계에서 자가 줄기세포 채취 및 이식 건수가 증가하고 있습니다.
• 특히 북미, 유럽, 중동에서의 보급에 기여한 것은 전략적 상환 노력과 지불자와의 협력입니다.

2. 주요 적응증 확대

• 겸상적혈구증(SCD) : CASGEVY는 12세 이상의 반복적인 혈관폐쇄성 위기(VOC) 환자들에게 승인되었으며, 태아 헤모글로빈 생성을 촉진하여 이러한 고통스러운 증상을 없애거나 크게 완화하는 데 도움을 줍니다.
• 수혈 의존성 베타 지중해빈혈(TDT) : 12세 이상의 적격 TDT 환자에게도 승인되어 수혈의 필요성을 크게 줄이거나 없앨 수 있는 치료법입니다.
• 소아 적응증 확대: 5-11세 소아를 대상으로 한 새로운 임상 데이터는 유망하며, 현재 진행 중인 전 세계 규제 당국 신청 및 적응증 확대 가능성의 토대를 형성하고 있습니다.
• 기능적 치료 가능성: CASGEVY의 CRISPR 유전자 편집 기술은 BCL11A 유전자의 적혈구 특이적 영역을 표적으로 하여 증상뿐만 아니라 근본적인 질병 메커니즘을 해결함으로써 태아 헤모글로빈의 재활성화를 실현합니다.

3. 지역적 확장

• CASGEVY의 규제 당국 승인은 미국, EU(조건부), 영국, 바레인, 사우디아라비아 등 중동 국가로 확대되어 환자 접근성을 확대하고 있습니다.
• 미국, 유럽, 중동 전역에 공인치료센터(ATC)가 지속적으로 개설되고 있으며, 환자의 치료 과정을 지원하는 인프라가 확충되고 있습니다.
• 향후 수년간, 더 많은 국가에서 자격을 갖춘 환자들을 지원하기 위해 보험 적용 범위를 확대하기 위한 노력을 계속할 것으로 예측됩니다.

4. 신규 적응증 승인

• SCD 및 TDT: CASGEVY는 2023년 12월 미국 FDA로부터 SCD에 대한 승인을 받았으며, 2024년 1월에는 TDT에 대한 승인도 받아 중요한 규제 마일스톤을 달성했습니다.
• EU의 승인도 2024년 초에 SCD와 TDT 모두에 대해 EU내 판매 승인이 확대되었습니다.
• 저연령층에서의 초기 임상 데이터를 바탕으로 소아 적응증에 대한 승인 신청이 전 세계에서 진행 중입니다.

5. 임상 사례의 견고한 증가 추세

• CLIMB 임상시험의 장기 데이터에 따르면 추적관찰 기간이 길어질수록 태아 헤모글로빈 수치가 지속적으로 상승하고 임상적 효과가 지속되는 것으로 나타났습니다.
• 치료를 받은 환자 중 높은 비율의 환자가 SCD의 경우 VOCs-free(수혈이 필요 없는 상태), TDT의 경우 수혈이 필요 없는 상태를 달성하여 임상적 가치를 입증하고 있습니다.
• 초기 임상 도입 데이터(2025년 3분기까지)에 따르면 전 세계에서 세포 채취 및 수혈 건수가 증가 추세에 있으며, 치료의 모멘텀이 증가하고 있는 것으로 나타났습니다.

6. 경쟁 차별화 및 시장 동향

• 단회 투여로 잠재적인 근본적 치료법: CASGEVY의 CRISPR/Cas9 편집 기술은 만성 치료법이나 지지요법에 비해 뚜렷한 우위를 가지고 있으며, 유전성 질환의 근본적 정밀의료의 추세에 부합합니다.
• 양호한 임상 프로파일: 태아 헤모글로빈의 지속적인 증가로 질병 관련 사건이 감소하고 있습니다. 임상시험에서 일관된 안전성 프로파일이 확인되었지만, 골수 파괴성 질환 치료에는 예상 가능한 위험이 수반됩니다.
• 맞춤형 의료와 유전자 편집 기술의 보급: 유전자 편집 기술의 광범위한 수용과 장기 추적 연구를 통한 근거가 지불 기관과 임상의의 신뢰를 지원하고 있습니다.
• 파이프라인의 혁신성: 차세대 생체내 편집 및 전처리 접근법에 대한 지속적인 연구는 유전자 치료의 전망을 더욱 넓힐 수 있습니다.

이 보고서는 미국, EU4(독일, 프랑스, 이탈리아, 스페인, 이탈리아, 스페인), 영국 등 주요 7개국(미국, EU4, 독일, 프랑스, 이탈리아, 스페인, 영국)의 베타 지중해빈혈 및 겸상적혈구증 등의 적응증에 대한 CASGEVY의 종합적인 인사이트를 제공합니다. 2020-2034년까지 케케묵은 주요 7개국에서 CASGEVY의 사용 현황, 진출 전망, 실적에 대한 상세한 전망을 제공하고, 승인된 적응증에 대한 CASGEVY의 상세한 설명을 제시합니다. 또한 이 약물의 매출 예측, 작용기전(MoA), 용량 및 투여방법, 규제 마일스톤을 포함한 연구개발 및 기타 활동에 대한 정보를 제공합니다. 또한 과거 및 현재 CASGEVY의 실적, 미래 시장 평가, SWOT 분석, 애널리스트의 견해, 경쟁사 개요, 각 적응증에 대한 다른 신흥 치료제의 개요, 매출 예측 분석, 시장 견인 요인 등을 정리했습니다.

목차

제1장 리포트 개요

제2장 CASGEVY 개요 : 베타지중해빈혈 및 겸상 적혈구증 등의 승인 적응증

제3장 CASGEVY : 경쟁 구도(출시 치료제)

제4장 CASGEVY : 경쟁 구도(개발 후기 치료제)

제5장 CASGEVY : 시장 평가

제6장 CASGEVY : SWOT 분석

제7장 애널리스트의 견해

제8장 부록

제9장 DelveInsight의 서비스 내용

제10장 면책사항

제11장 DelveInsight 소개

제12장 리포트 구입 옵션

영문목차

Key Factors Driving CASGEVY Growth

1. Market Share Gains and New Patient Starts

• CASGEVY is rapidly gaining adoption in the rare hematology market, especially in sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT), due to its one-time, potentially curative profile compared to lifelong supportive care.
• Expanded eligibility and growing treatment center activation are fueling patient starts, with increasing numbers of autologous stem-cell collections and infusions reported globally.
• Strategic reimbursement efforts and payer engagement are helping uptake, particularly across North America, Europe and the Middle East.

2. Expansion Across Key Indications

• Sickle Cell Disease (SCD): CASGEVY is approved for patients aged 12 years and older with recurrent vaso-occlusive crises (VOCs), offering elimination or substantial reduction of these painful events through enhanced fetal hemoglobin production.
• Transfusion-Dependent Beta Thalassemia (TDT): The therapy is also approved for eligible TDT patients aged 12 years and older, significantly reducing or eliminating transfusion requirements.
• Pediatric Expansion: Emerging clinical data for ages 5-11 are promising and form the basis for ongoing global regulatory submissions and potential label expansions.
• Functional Cure Potential: CASGEVY's CRISPR gene editing targets the erythroid-specific region of the BCL11A gene, reactivating fetal hemoglobin and addressing underlying disease mechanisms rather than only symptoms.

3. Geographic Expansion

• CASGEVY's regulatory approvals span the United States, European Union (conditional), United Kingdom, and Middle Eastern countries such as Bahrain and Saudi Arabia, broadening patient access.
• Authorized Treatment Centers (ATCs) continue to be activated across the US, Europe and Middle East, increasing infrastructure to support patient treatment journeys.
• Ongoing efforts to expand reimbursement access aim to support eligible patients in more countries over the coming years.

4. New Indication Approvals

• SCD & TDT: CASGEVY received US FDA approval for SCD in December 2023 and later for TDT in January 2024, marking significant regulatory milestones.
• European Commission authorization followed in early 2024 for both SCD and TDT, extending its EU marketing status.
• Pediatric regulatory submissions are underway globally, driven by early clinical data in younger age groups.

5. Strong Clinical Volume Momentum

• Long-term data from CLIMB clinical trials show durable increases in fetal hemoglobin levels and sustained clinical benefit with extended follow-up.
• High percentages of treated patients achieve VOCs freedom in SCD and transfusion independence in TDT, reinforcing clinical value.
• Early real-world launch data (through third quarter 2025) indicate a growing number of cell collections and infusions globally, signaling an increasing treatment momentum.

6. Competitive Differentiation and Market Trends

• One-time, potentially curative therapy: CASGEVY's CRISPR/Cas9 editing offers a distinct advantage over chronic therapies and supportive care approaches, aligning with the trend toward curative precision medicines in genetic disorders.
• Favorable clinical profiles: Durable increases in fetal hemoglobin reduce disease-related events with a consistent safety profile observed in trials, though myeloablative conditioning carries expected risks.
• Personalized medicine & gene editing adoption: Broader acceptance of gene editing and evidence from long-term follow-ups support payer and clinician confidence.
• Pipeline innovation: Ongoing research into next-generation in vivo editing and conditioning approaches could further expand the gene-therapy landscape.

CASGEVY Recent Developments

• FDA approvals of CASGEVY for SCD (Dec 2023) and TDT (Jan 2024) marked historic milestones as the first CRISPR/Cas9 gene-editing therapies commercially authorized in the US.
• The European Commission granted conditional authorization for both indications in early 2024.
• Positive long-term clinical data demonstrating consistent and durable benefits were presented at major hematology congresses (EHA, ASH).
• New pivotal data from pediatric cohorts (ages 5-11) were shared at the ASH Annual Meeting, supporting expanded regulatory filings.

"CASGEVY Sales Forecast, and Market Size Analysis - 2034" report provides comprehensive insights of CASGEVY for approved indication like Beta-thalassaemia and Sickle cell anaemia in the 7MM. A detailed picture of CASGEVY's existing usage in anticipated entry and performance in approved indications in the 7MM, i.e., the United States, EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan for the study period 2020 -2034 is provided in this report along with a detailed description of the CASGEVY for approved indications. The CASGEVY market report provides insights about CASGEVY's sales forecast, mechanism of action (MoA), dosage and administration, as well as research and development including regulatory milestones, along with other developmental activities. Further, it also consists of historical and current CASGEVY performance, future market assessments inclusive of the CASGEVY market forecast analysis for approved indications in the 7MM, SWOT, analysts' views, comprehensive overview of market competitors, and brief about other emerging therapies in respective indications. It also provides analysis of CASGEVY sales forecasts, along with factors driving its market.

CASGEVY Drug Summary

CASGEVYZ (Exagamglogene autotemcel) is an autologous CRISPR/Cas9 genome-edited hematopoietic stem cell therapy designed to treat sickle cell disease (SCD) in patients 12 years and older with recurrent vaso-occlusive crises (VOCs), as well as transfusion-dependent B-thalassemia (TDT) in patients 12 years and older. It involves editing the patient's own CD34+ hematopoietic stem cells (HSPCs) ex vivo at the BCL11A gene's erythroid-specific enhancer to reduce BCL11A expression, thereby reactivating fetal hemoglobin (HbF) production; post-infusion, these edited cells engraft in the bone marrow, differentiate into erythroid lineage cells, increase Y-globin and HbF levels, reduce intracellular sickle hemoglobin (HbS) concentration, prevent red blood cell sickling, eliminate VOCs in SCD, and enable transfusion independence in TDT. Administered as a one-time intravenous infusion following myeloablative conditioning, mobilization, apheresis, and manufacturing, CASGEVY represents a personalized gene therapy given in specialized centers by trained physicians. The report provides CASGEVY's sales, growth barriers and drivers, post usage and approvals in multiple indications.

Scope of the CASGEVY Market Report

The report provides insights into:

• A comprehensive product overview including the CASGEVY MoA, description, dosage and administration, research and development activities in approved indication like Beta-thalassaemia and Sickle cell anaemia.
• Elaborated details on CASGEVY regulatory milestones and other development activities have been provided in CASGEVY market report.
• The report also highlights CASGEVY's cost estimates and regional variations, reported and estimated sales performance, research and development activities in approved indications across the United States, Europe, and Japan.
• The CASGEVY market report also covers the patents information, generic entry and impact on cost cut.
• The CASGEVY market report contains current and forecasted CASGEVY sales for approved indications till 2034.
• Comprehensive coverage of the late-stage emerging therapies for respective indications.
• The CASGEVY market report also features the SWOT analysis with analyst views for CASGEVY in approved indications.

Methodology:

The CASGEVY market report is built using data and information sourced primarily from internal databases, primary and secondary research and in-house analysis by DelveInsight's team of industry experts. Information and data from the secondary sources have been obtained from various printable and nonprintable sources like search engines, news websites, global regulatory authorities websites, trade journals, white papers, magazines, books, trade associations, industry associations, industry portals and access to available databases.

CASGEVY Analytical Perspective by DelveInsight

• In-depth CASGEVY Market Assessment

This CASGEVY sales market forecast report provides a detailed market assessment of CASGEVY for approved indication like Beta-thalassaemia and Sickle cell anaemia in the seven major markets, i.e., the United States, EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan. This segment of the report provides current and forecasted CASGEVY sales data uptil 2034.

• CASGEVY Clinical Assessment

The CASGEVY market report provides the clinical trials information of CASGEVY for approved indications covering trial interventions, trial conditions, trial status, start and completion dates.

CASGEVY Competitive Landscape

The report provides Insights on competitors and marketed products within the domain, along with a summary of emerging products and their respective launch dates, posing significant competition in the market.

CASGEVY Market Potential & Revenue Forecast

• Projected market size for the CASGEVY and its key indications
• Estimated CASGEVY sales potential (CASGEVY peak sales forecasts)
• CASGEVY Pricing strategies and reimbursement landscape

CASGEVY Competitive Intelligence

• Number of competing drugs in development (pipeline analysis)
• CASGEVY Market positioning compared to existing treatments
• CASGEVY Strengths & weaknesses relative to competitors

CASGEVY Regulatory & Commercial Milestones

• CASGEVY Key regulatory approvals & expected launch timelines
• Commercial partnerships, licensing deals, and M&A activity

CASGEVY Clinical Differentiation

• CASGEVY Efficacy & safety advantages over existing drugs
• CASGEVY Unique selling points

CASGEVY Market Report Highlights

• In the coming years, the CASGEVY market scenario is set to change due to strong adoption, increased prescriptions and broader uptake in multiple immunological indications; which would expand the size of the market.
• The CASGEVY companies are developing therapies that focus on novel approaches to treat/improve the disease condition, assess challenges, and seek opportunities that could influence CASGEVY's dominance.
• Other emerging products for Beta-thalassaemia and Sickle cell anaemia are expected to give tough market competition to CASGEVY and launch of late-stage emerging therapies in the near future will significantly impact the market.
• A detailed description of regulatory milestones, and developmental activities, provide the current development scenario of CASGEVY in approved indications.
• Analyse CASGEVY cost, pricing trends and market positioning to support strategic decision-making in the immunology landscape.
• Our in-depth analysis of the forecasted CASGEVY sales data uptil 2034 will support the clients in decision-making process regarding their therapeutic portfolio by identifying the overall scenario of CASGEVY in approved indications.

Key Questions:

• What is the class of therapy, route of administration and mechanism of action of CASGEVY? How strong is CASGEVY's clinical and commercial performance?
• What is CASGEVY's clinical trial status in each individual indications such as Beta-thalassaemia and Sickle cell anaemia and study completion date?
• What are the key collaborations, mergers and acquisitions, licensing and other activities related to the CASGEVY Manufacturers?
• What are the key designations that have been granted to CASGEVY for approved indications? How are they going to impact CASGEVY's penetration in various geographies?
• What is the current and forecasted CASGEVY market scenario for approved indications? What are the key assumptions behind the forecast?
• What are the current and forecasted sales of CASGEVY in the seven major countries, including the United States, Europe (Germany, France, Italy, Spain) and the United Kingdom, and Japan?
• What are the other emerging products available and how are these giving competition to CASGEVY for approved indications?
• Which are the late-stage emerging therapies under development for the treatment of approved indications?
• How cost-effective is CASGEVY? What is the duration of therapy and what are the geographical variations in cost per patient?

Table of Contents

1. Report Introduction

2. CASGEVY Overview in approved indications like Beta-thalassaemia and Sickle cell anaemia

• 2.1. Product Detail
• 2.2. CASGEVY Clinical Development
  • 2.2.1. CASGEVY Clinical studies
  • 2.2.2. CASGEVY Clinical trials information
  • 2.2.3. Safety and efficacy
• 2.3. Other Developmental Activities
• 2.4. Product Profile

3. CASGEVY Competitive Landscape (Marketed Therapies)

4. Competitive Landscape (Late-stage Emerging CASGEVY Therapies)

5. CASGEVY Market Assessment

• 5.1. CASGEVY Market Outlook in approved indications
• 5.2. 7MM Analysis
  • 5.2.1. CASGEVY Market Size in the 7MM for approved indications
• 5.3. Country-wise Market Analysis
  • 5.3.1. CASGEVY Market Size in the United States for approved indications
  • 5.3.2. CASGEVY Market Size in Germany for approved indications
  • 5.3.3. CASGEVY Market Size in France for approved indications
  • 5.3.4. CASGEVY Market Size in Italy for approved indications
  • 5.3.5. CASGEVY Market Size in Spain for approved indications
  • 5.3.6. CASGEVY Market Size in the United Kingdom for approved indications
  • 5.3.7. CASGEVY Market Size in Japan for approved indications

6. CASGEVY SWOT Analysis

7. Analysts' Views

8. Appendix

• 8.1. Bibliography
• 8.2. Report Methodology

9. DelveInsight Capabilities

10. Disclaimer

11. About DelveInsight

12. Report Purchase Options