Aplastic Anemia Treatment Market Size, Emerging Drugs, Market Outlook, and Companies 2034

DelveInsight's "Aplastic Anemia Market Insights, Epidemiology and Market Forecast - 2034" report delivers an in-depth understanding of the aplastic anemia, historical and forecasted epidemiology as well as the aplastic anemia market trends in the United States, EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan.

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Key Takeaways from the Aplastic Anemia Market Report
• In October 2024:- Novartis Pharmaceuticals-This is a phase II, open label, multi-center, intra-patient dose escalation study to characterize the pharmacokinetics (PK) after oral administration of eltrombopag in combination with immunosuppressive therapy in pediatric patients with previously untreated or relapsed/refractory severe aplastic anemia or recurrent aplastic anemia.
• In October 2024:- This study is researching an experimental drug called REGN7257 (called "study drug"). The study is focused on patients who have severe aplastic anemia (SAA), a disease of the bone marrow resulting in an impairment of the production of blood cells. The main purpose of this two-part study (Part A and Part B) is to test how safe and tolerable REGN7257 is in patients with SAA in which other Immunosuppressive therapies (ISTs) have not worked well.
• The increase in Aplastic Anemia market size is a direct consequence of high incidence in the 7MM.
• The total Aplastic Anemia incident cases in the 7MM comprised approximately ~2,500 cases in 2023 and are projected to increase during the forecast period.
• Among the 7MM, the Japan accounted for the highest number of Aplastic Anemia incident cases i.e., ~800 cases in 2023.
• The leading Aplastic Anemia Companies working in the market include Pfizer, BioLineRx, Ltd., Regeneron Pharmaceuticals, Gamida Cell, Elixirgen, Hangzhou Zede Pharmaceutical Technology, Cellenkos, Hemogenyx Pharmaceuticals, and others.
• Promising Aplastic Anemia Pipeline Therapies in the various stages of development include Romiplostim, PF-06462700, Eltrombopag 25 mg, Eltrombopag, hATG, CsA, Cyclosporine, AMG531 and others.

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Aplastic Anemia Epidemiology Segmentation in the 7MM
• Total Aplastic Anemia Incidence Cases
• Aplastic Anemia Age-related cases
• Aplastic Anemia Severity- specific Incident Cases

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Aplastic Anemia Market Insights
Management of aplastic anemia depends on age, disease severity, donor availability, and performance status. The treatment aims to correct the bone marrow failure, as well as to treat the patient's immediate signs and symptoms. The two main forms of specific treatment are bone marrow transplantation and immunosuppressive therapies. Young patients (younger than 50 years) are advised to undergo allogeneic hematopoietic cell transplant (HCT) before initial immunosuppressive therapy. Old and young patients without an HCT donor receive full-dose immunosuppressive therapy using eltrombopag, horse/rabbit anti-thymocyte globulin (ATG), cyclosporine A, and prednisone. This combination can be tailored to single-agent eltrombopag, ATG, or cyclosporine A for less healthy individuals.

Aplastic Anemia Marketed Drugs
• PROMACTA/REVOLADE (eltrombopag): Novartis
PROMACTA/REVOLADE (eltrombopag) tablets contain eltrombopag olamine, a small molecule thrombopoietin (TPO) receptor agonist for oral administration. Eltrombopag, sold under the brand name PROMACTA, among others, is a medication used to treat thrombocytopenia and severe aplastic anemia (SAA). Eltrombopag is sold under the brand name REVOLADE outside the US and is marketed by Novartis. In August 2014, the US FDA approved a sNDA for the once-daily use of PROMACTA in patients with severe aplastic anemia (SAA) who had an insufficient response to immunosuppressive therapy.

• ROMIPLATE (romiplostim; AMG531): Kyowa Kirin/Amgen
ROMIPLATE is composed of recombinant protein acting on the thrombopoietin receptor, which has been licensed from Amgen (K-A) to Kyowa Kirin. It was launched as a drug for idiopathic thrombocytopenic purpura (ITP) in April 2011 and for aplastic anemia in patients who had an inadequate response to conventional therapy in June 2019 in Japan. The drug is composed of recombinant protein stimulating hematopoiesis via acting on the thrombopoietin receptors. It acts directly on megakaryocytic progenitor cells and exerts platelet hematopoietic effects.

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Aplastic Anemia Emerging Drugs
• REGN7257: Regeneron Pharmaceuticals
REGN7257 is a gc cytokine receptor antibody that targets the common g chain (gc; IL-2RG) found in interleukin (IL) receptors for various gc cytokines, including IL-2, IL-4, IL-7, IL-9, IL-15, and IL-21. It was developed to investigate the potential of targeting gc cytokines for the prevention and treatment of T-cell-mediated diseases. By blocking the signaling of gc cytokines, REGN7257 aims to ameliorate T-cell-mediated pathogenesis. The drug REGN7257 is presently undergoing evaluation in a Phase I/II clinical trial to assess its safety and tolerability in patients with severe aplastic anemia (SAA) who have either relapsed after immunosuppressive therapy (IST) or are refractory to IST.

• OMISIRGE (omidubicel): Gamida Cell
OMISIRGE (omidubicel-onlv) is a cryopreserved nicotinamide-modified allogeneic hematopoietic progenitor cell therapy derived from cord blood and manufactured utilizing a proprietary NAM-based technology producing enriched HPCs. It is indicated for use in adults and pediatric patients aged 12 years and older with hematologic malignancies who are planned for umbilical cord blood transplantation following myeloablative conditioning. Omisirge has received approval for use in hematological malignancies and is currently undergoing evaluation in an ongoing investigator-sponsored Phase I/II study focusing on patients with severe aplastic anemia.

Aplastic Anemia Drug Market Landscape
The prognosis for patients with Aplastic anemia has markedly improved because of better treatment and supportive measures. Depending on the cause, with treatment, the outcomes of hematopoietic cell transplantations are excellent. With the use of immunosuppressive therapy, there is a risk of relapse and late clonal disease. Despite being efficacious in inducing and maintaining remission in organ threatening inflammatory diseases, Immunosuppressive drugs are also associated with significant adverse effects and toxicity. Current treatments have been moderately successful in reducing the severity of Aplastic anemia in patients.

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Aplastic Anemia Treatment Options
• Promacta (Novartis)
• Aranesp (Kirin-Amgen)
• CellCept (Roche)
• Cytoxan (Hikma Pharmaceuticals)

Aplastic Anemia Therapeutics Market
Immunosuppressive drugs has limitations for its use as they suppress the immune system and are inevitably associated with increased risk of infection and malignancy. Many of these drugs also impact adversely and increases cardiovascular risk. The risk of infection increases with the degree of immunosuppression. Infections with Pneumocystis jirovecii, nocardia, aspergillus, Cryptococcus and reactivation of varicella zoster, herpes simplex, cytomegalovirus, hepatitis B and C as well as tuberculosis are common and the risk of cancer, especially cutaneous and hematological malignancies are also increased.

Aplastic Anemia Drugs Uptake
• REGN7257 (Regeneron Pharmaceuticals) is an anti-interleukin 2 receptor subunit gamma [IL2RG] monoclonal antibody. Currently, it is in Phase I/II of clinical trials to assess the safety and tolerability of REGN7257 in patients with immunosuppressive therapy (IST)-refractory or IST-relapsed severe aplastic anemia (SAA). An additional primary objective is to evaluate the clinical efficacy of REGN7257 in patients with IST-refractory or IST-relapsed SAA.

• Omidubicel (Gamida-Cell) is an advanced cell therapy under development as a potential life-saving allogeneic hematopoietic stem cell transplant solution for patients with hematologic malignancies. Omidubicel is the first bone marrow transplant product to receive Breakthrough Therapy Designation from the US FDA and has also received Orphan Drug Designation in the US and EU. In June 2022, the company announced completion of the rolling Biologics License Application (BLA) submission to the US FDA for omidubicel for the treatment of patients with blood cancers in need of an allogenic hematopoietic stem cell transplant. Currently, omidubicel is being evaluated in an ongoing investigator-sponsored Phase 1/2 study in patients with severe aplastic anemia.

• Romiplostim (Kyowa Kirin) is a thrombopoietin receptor agonist that binds to and activates the TPO receptor on megakaryocyte precursors, thus promoting cell proliferation and viability, resulting in increased platelet production. In July 2018, the company announced seeking approval of an additional indication for romiplostim (code name: AMG531, "the drug") for aplastic anemia (AA) has been filed with Japan's Ministry of Health, Labour and Welfare (MHLW).

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Scope of the Aplastic Anemia Market Report
• Coverage- 7MM
• Study Period- 2020-2034
• Aplastic Anemia Companies- Pfizer, BioLineRx, Ltd., Regeneron Pharmaceuticals, Gamida Cell, Elixirgen, Hangzhou Zede Pharmaceutical Technology, Cellenkos, Hemogenyx Pharmaceuticals, and others.
• Aplastic Anemia Pipeline Therapies- Romiplostim, PF-06462700, Eltrombopag 25 mg, Eltrombopag, hATG, CsA, Cyclosporine, AMG531 and others.
• Aplastic Anemia Market Dynamics: Aplastic Anemia Market Drivers and Barriers
• Aplastic Anemia Market Access and Reimbursement & Unmet Needs

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Table of Content
1. Key Insights
2. Report Introduction
3. Aplastic Anemia Executive Summary
4. Aplastic Anemia Market Overview at a Glance
5. Key Events
6. Aplastic Anemia Epidemiology and Market Forecast Methodology
7. Disease Background and Overview
8. Treatment and Management of Aplastic Anemia
9. Aplastic Anemia Epidemiology and Patient Population
10. Patient Journey
11. Aplastic Anemia Marketed Therapies
12. Aplastic Anemia Emerging Drugs
13. Retinitis Pigmentosa: Market Analysis
14. Aplastic Anemia Unmet Needs
12. Aplastic Anemia SWOT Analysis
13. Aplastic Anemia KOL Views
14. Aplastic Anemia Market Access and Reimbursement
15. Appendix
16. DelveInsight Capabilities
17. Disclaimer
18. About DelveInsight

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