US Sickle Cell Disease Drugs Market Analysis

US Sickle Cell Disease Drugs Market Analysis

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US Sickle cell disease Drugs market is projected to grow from $xx Mn in 2023 to $xx Mn by 2030, registering a CAGR of xx% during the forecast period of 2023 - 2030. The market for Sickle cell disease Drugs is growing rapidly as a result of increasing prevalence of sickle cell disease , increasing investment for healthcare infrastructure, increase in the number of research and development activities, Support provided by governments of different countries, patient advocacy programmes and incentives provided by regulatory authorities. Global Blood Therapeutics, Novartis, Emmaus Life Sciences, Bluebird Bio, Teva Pharmaceutical Industries Ltd, Pfizer Inc., Mylan N.V., Merck & Co., Inc., Johnson & Johnson Private Limited, Bristol-Myers Squibb Company, AbbVie Inc are the key global market players operating in sickle cell disease Drugs market.

ID: IN10USRD112 CATEGORY: Rare Diseases GEOGRAPHY: US AUTHOR: Gaurav Bhongale

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US Sickle Cell Disease Drugs Market Analysis Summary

US Sickle Cell Disease Drugs Market is valued at around $1 Bn in 2022 and is projected to reach $2.24 Bn by 2030, exhibiting a CAGR of 10.6% during the forecast period 2023-2030.

Sickle cell disease (SCD) is a genetic blood disorder characterized by the presence of abnormal haemoglobin that causes red blood cells to become rigid and sickle-shaped. It happens due to a mutation on the sixth position of the beta chain of hemoglobin where glutamic acid amino acid gets converted into valine. Bone marrow transplantation is the only permanent cure for SCD, it is usually reserved for individuals under the age of 16 since bone marrow transplant risk in adults is too great. Symptomatic relief and palliative care are also done for this disease. Hydroxyurea and Endari are the only two licensed medicines available for this disease. Global Blood Therapeutics, Novartis, Emmaus Life Sciences, Bluebird Bio, Teva Pharmaceutical Industries Ltd, Pfizer Inc., Mylan N.V., Merck & Co., Inc., Johnson & Johnson Private Limited, Bristol-Myers Squibb Company, AbbVie Inc are the key global market players operating in sickle cell disease Drugs market.

Market Dynamics

Market Drivers

Increasing prevalence of sickle cell disease.

Increasing investment in healthcare infrastructure

Increase in the number of research and development activities, Support provided by governments of different countries

Patient advocacy programmes and incentives provided by regulatory authorities like orphan drug designations. All these factors act as market growth drivers.

Market Restraints

High cost associated with treatment, lack of skilled professionals and physicians for treatment, lack of reimbursement policies and skilled professionals for treatment. All these act as restraints of sickle cell disease drug market development.

Notable deals in the Sickle cell disease Drugs market

In December 2021, Oxbryta (voxelotor) was approved by US FDA for treating sickle cell disease in patients aged 4 up to 11 years. 

In May 2022, multiple daily doses of GBT601, Global Blood Therapeutics (GBT) experimental oral therapy for sickle cell disease (SCD), were well tolerated and showed promising pharmacological and efficacy signals in six patients in a phase I trial clinical trial.

Key players

Global Blood Therapeutics, Inc. Novartis AG Pfizer Inc. Bluebird Bio, Inc. Emmaus Life Sciences, Inc. Sanofi Genzyme Mast Therapeutics, Inc. Imara Inc. Vertex Pharmaceuticals Incorporated Global Blood Therapeutics, Inc.

1. Executive Summary
1.1 Disease Overview
1.2 Global Scenario
1.3 Country Overview
1.4 Healthcare Scenario in Country
1.5 Patient Journey
1.6 Health Insurance Coverage in Country
1.7 Active Pharmaceutical Ingredient (API)
1.8 Recent Developments in the Country

2. Market Size and Forecasting
2.1 Epidemiology of Disease
2.2 Market Size (With Excel & Methodology)
2.3 Market Segmentation (Check all Segments in Segmentation Section)

3. Market Dynamics
3.1 Market Drivers
3.2 Market Restraints

4. Competitive Landscape
4.1 Major Market Share

4.2 Key Company Profile (Check all Companies in the Summary Section)

4.2.1 Company Overview Product Applications and Services Recent Developments Partnerships Ecosystem Financials (Based on Availability)

5. Reimbursement Scenario
5.1 Reimbursement Regulation
5.2 Reimbursement Process for Diagnosis
5.3 Reimbursement Process for Treatment

6. Methodology and Scope

Market Segmentations For US Sickle cell disease Drugs market

By Type

  • Sickle Cell Anemia
  • Sickle Hemoglobin- C Disease
  • Sickle Beta-Plus Thalassemia
  • Sickle Hemoglobin-D Disease
  • Sickle Hemoglobin-O Disease
  • Others

By Diagnosis

  • Screening Tests
  • New-born Screening
  • Prenatal Screening
  • Others

By Treatment

  • Disease Modifying Therapies
  • Gene therapies
  • Gene editing therapies
  • Targeted therapies
  • Symptomatic Treatments
  • Hydroxyurea
  • Blood Transfusion
  • Bone Marrow Transplantation
  • Penicillin
  • Analgesics

By Route of Administration

  • Oral
  • Parenteral
  • Others

By End-Users

  • Hospitals
  • Specialty Clinics
  • Homecare
  • Others

By Distribution Channel

  • Hospital Pharmacy
  • Retail Pharmacy
  • Online Pharmacy
  • Others

Insights10 will provide you with the reports within 10 key parameters which are:

  1. Market Overview
  2. Market Growth Drivers & Restraints
  3. Epidemiology of Disease Type
  4. Market Segmentation
  5. Market Share
  6. Competitive Landscape
  7. Key Company Profiles
  8. Healthcare Policies & Regulatory Framework
  9. Reimbursement Scenario
  10. Factors Driving Future Growth

Based on our many years of experience, we believe that these are the parameters that are critical to decision-making for business stakeholders. Our focused approach to developing reports focused on 10 key parameters, enabled us to arrive at the name “Insights10”.


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The study on the market covers the analysis of the leading geographies such as Asia-Pacific, Africa, Europe, Middle East, North America, and  Latin America for the period of 2022 to 2030. The qualitative analysis covers the industry landscape and trends, market opportunities, competitive landscape, and policy and regulatory scenario, and the quantitative analysis covers different market estimates and forecasts.

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Stage III: Interpretation and Presentation

Analysis & Interpretation: The information gathered is then analyzed and synthesized. The second series of interviews are done if necessary to check and validate. The future opportunities are analyzed by understanding product commercialization and many other factors. It also comprises the analysis of data discrepancies observed across various data sources. Information procured from secondary and primary results is then, interpreted by considering the following parameters: (a partial list)

  • Establishing market drivers and trends
  • Analyzing the regulatory landscape to understand future growth
  • Market Segment based analysis to obtain revenue/volume
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The foreign exchange rates are calculated on the respective dates and for the respective regions covered in the study.

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Last updated on: 04 July 2023
Updated by: Dhruv Joshi

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