CASGEVY Sales Forecast, and Market Size Analysis - 2034

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?