Sickle Cell Anaemia - Drug Pipeline Landscape, 2023

Sickle Cell Anaemia - Drug Pipeline Landscape, 2023

Sickle Cell Anaemia is a form of the inherited blood disorder, called as sickle cell disease which changes the shape of red blood cells, turns round flexible discs into stiff, rigid and sticky sickle cells that blocks blood flow and oxygen to all parts of the body. As a result, there are low healthy red blood cells leads to develop Anaemia, hence the condition is Sickle Cell Anemia.

The most common cause of Sickle Cell Anaemia is inheritance of the disease from the biological parents or changes in the gene which produces the haemoglobin, the iron-rich compound in red blood cells.

Symptoms include Anaemia, pain episodes, infections, swelling, delayed growth and vision related issues.

Doctors most often diagnose Sickle Cell Anaemia by blood tests, prenatal genetic screening.

The most common treatments for Sickle Cell Anemia are medications aimed to avoid pain episodes and prevents complications, blood transfusions and stem cell transplantation.

Major pharmaceutical companies are involved in the development of potential drug candidates to improve the Sickle Cell Anaemia treatment such as Exagamglogene Autotemcel, Etavopivat, Mitapivat, GBT021601, Lovotibeglogene Autotemcel and others. Key players involved in the development of therapies to treat sickle cell anaemia are Novo Nordisk A/S, Pfizer, Bluebird Bio, Vertex Pharmaceuticals Incorporated, Agios Pharmaceuticals Inc and others. One drug is under late-stage Pre-Registration, five drugs are under Phase III clinical trials, seventeen drugs are in Phase II clinical trials and some other drugs are under Phase I, IND/CTA Filed, Preclinical and Discovery stages of development.

In December 2022, Vertex Pharmaceuticals Incorporated announced that the rolling Biologics License Application (BLA) submission for Exagamglogene Autotemcel to the U.S. FDA has been initiated and plan to complete the BLA by the end of the first quarter 2023 and remain on track to submit exa-cel to the Medicines and Healthcare products Regulatory Agency in the U.K. and the EMA in the European Union by the end of the year.

In December 2022, Bluebird Bio Inc announced that the U.S. Food and Drug Administration (FDA) lifted its partial clinical hold for patients under the age of 18 in studies evaluating lovotibeglogene autotemcel (lovo-cel) for sickle cell disease and presented new data from a detailed investigation of cases at the American Society of Hematology (ASH) Annual Meeting and Exposition.

In November 2022, Agios Pharmaceuticals Inc published Mitapivat’s clinical and translational data supporting the utility of PK activators in sickle cell disease.

Report Highlights

Global Insight Service's, Sickle Cell Anemia - Drug Pipeline Landscape, 2023 report provides an overview of the Sickle Cell Anemia pipeline drugs. This report covers detailed insights on Sickle Cell Anemia drugs under development, assessment by target, mechanism of action, route of administration and molecule type. Product pipeline by companies, stage of development and key regulatory designations, deals and milestones have been presented to provide insights and thus help industry participants in their decision making. Sickle Cell Anemia pipeline report helps gain insights on drugs which are under development stage of drug development process across globally.

Methodology

The research process includes extensive secondary research on public domain and other authentic sources to add or update the pipeline products information. The secondary research sources include, but are not limited to company websites, annual reports, financial reports, company pipeline chart, investor presentations and SEC filings, journals and conferences, and clinical trials registries.

Scope

The pipeline landscape report provides analysis of pipeline products based on several stages of development ranging from Discovery to Pre-Registration. The report provides a review of pipeline therapeutics by companies based on information derived from company and industry-specific sources. The pipeline report covers assessment of therapeutics by mechanism of action (MoA), drug target, route of administration (RoA) and molecule type. Comprehensive profiles of the pipeline products with details such as company overview, development stage; molecule type, target, mechanism of action, route of administration, dosage form, regulatory designations, key deals, clinical trials, and key upcoming milestones are included.

Reasons to Buy
Helps to find and recognize significant therapeutics under development. Thorough understanding of pipeline structure and helps in developing corrective measures for pipeline projects.
Effective R&D strategies can be developed with deep knowledge of competitor information, analysis, and insights.
Plan collaborations with various industry partners that have role in some or the other stage of drug development such as contract manufacturing, co-development, contract research organization and commercialization etc.
Helps to create in-licensing and out-licensing opportunities by identifying prospective partners with attractive projects to expand business potential and scope.

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