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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.
The aplastic anemia market report provides current treatment practices, emerging drugs, market share of individual therapies, and current and forecasted 7MM aplastic anemia market size from 2020 to 2034. The report also covers current aplastic anemia treatment practices/algorithms and unmet medical needs to curate the best opportunities and assess the market's potential.
Study Period: 2020-2034
Aplastic Anemia Overview
Aplastic anemia refers to the syndrome of chronic primary hematopoietic failure from injury leading to diminished or absent hematopoietic precursors in the bone marrow and attendant pancytopenia. The symptoms of aplastic anemia occur because the bone marrow fails to produce enough blood cells. Some individuals may have mild symptoms that remain stable for many years; others may have serious symptoms that can progress to life-threatening complications. Individuals with anemia may experience tiredness, increased need for sleep, weakness, lightheadedness, dizziness, irritability, headaches, pale skin color, difficulty breathing, and cardiac symptoms like chest pain.
Aplastic Anemia Diagnosis
The rarity of aplastic anemia, coupled with its overlap with other bone marrow failure syndromes, poses a diagnostic challenge.Except for findings related to bleeding or infections, the examination presents mainly negative characteristics: absence of lymphadenopathy, no enlarged spleen or liver, and no infiltration of any other organ. Bone marrow (BM) aspiration and biopsy are essential procedures for diagnosing aplastic anemia. Cytogenetic abnormalities can be detected in up to 12-15% of otherwise typical aplastic anemia patients, making systematic cytogenetic investigations essential for all aplastic anemia patients. Molecular analysis, including next-generation sequencing (NGS), has become increasingly instrumental in comprehending the pathophysiology of diseases.
Aplastic Anemia Treatment
Treatment of aplastic anemia varies, depending upon the individual's age, general health, and the severity of aplastic anemia. 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. Initial treatment of acquired aplastic anemia may be directed toward improving the symptoms that may result from low blood counts. Such treatment consists of giving red blood cell transfusions to correct anemia, platelet transfusions to treat or prevent serious bleeding, and antibiotics to treat or prevent infections. Supportive care is a critical component in the management of aplastic anemia, aiming to address symptoms, complications, and overall patient well-being. Patients often require regular blood transfusions to alleviate anemia and manage low platelet counts.
Further details related to diagnosis and treatment are provided in the report…
As the market is derived using a patient-based model, the aplastic anemia epidemiology chapter in the report provides historical as well as forecasted epidemiology segmented by total incident cases of aplastic anemia, severity-specific cases of aplastic anemia, and age-specific cases of aplastic anemia in the 7MM covering the United States, EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan from 2020 to 2034.
The drug chapter segment of the aplastic anemia report encloses a detailed analysis of aplastic anemia marketed drugs and emerging pipeline drugs. It also helps understand the aplastic anemia pivotal clinical trial details, recent and expected market approvals, patent details, each drug's advantages and disadvantages, the latest news, and recent deals and collaborations.
Marketed Drug
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. And later in November 2018, the us FDA expanded the label for PROMACTA (eltrombopag) to include first-line treatment for adults and pediatric patients 2 years and older with SAA in combination with IST.
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.
Emerging Drug
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.
In real-world scenarios, the approach to treating NSAA differs significantly from SAA/VSAA. Unlike SAA/VSAA, hematopoietic stem cell transplantation (HSCT) is not employed in NSAA cases. The criteria for determining when to initiate treatment for NSAA are less clearly defined, and there is a lack of comprehensive recommendations for clinical evaluation and therapeutic strategies. The ultimate management of NSAA predominantly relies on the expertise of individual medical centers. Historically, the primary modalities for treating aplastic anemia have been IST and BMT in eligible patients. For individuals with SAA/VSAA deemed suitable for transplant-based interventions, age emerges as a pivotal determinant influencing survival post-matched sibling donor (MSD) allogeneic transplantation. Notably, older patients, often having undergone prior IST, presenting with additional comorbidities, diminished performance status, and an extended duration between diagnosis and BMT, exhibit distinctive challenges in outcomes, with fludarabine-containing regimens offering more promising results in this demographic, albeit based on limited retrospective analyses. In the realm of second-line treatments, patients with aplastic anemia are considered eligible for transplantation in cases of refractory status following first-line IST. IST in the form of horse ATG + CyA has been extensively researched regimen in Europe, Asia, and the United States over several decades. Recent endeavors to enhance the efficacy of horse ATG have involved augmenting immunosuppression with agents such as mycophenolate, sirolimus, rabbit ATG, alemtuzumab, and cyclophosphamide.
Approximately a decade ago, notable progress was made with the approval of thrombopoietin receptor agonists for immune thrombocytopenia, effectively inducing platelet count recovery in refractory cases. Distinguishing itself from erythropoietin and G-CSF, the Tpo receptor is expressed in stem cells. Tpo receptor knockout models revealed significant reductions not only in megakaryocytes and platelets but also in HSCs. In 2014, the US FDA approved PROMACTA for the treatment of aplastic anemia, which then approved in Europe and Japan in 2015 and 2017, respectively, under the brand name REVOLADE. Later, another Tpo receptor agonist, ROMIPLATE, was granted approval in Japan in June 2019 for aplastic anemia cases where conventional treatments have proven insufficient.
With respect to the emerging pipeline, there is a scarcity of advancements in SAA treatment, with only two companies in the 7MM actively engaged in developing therapies-Regeneron Pharmaceuticals with REGN7257 and Gamida Cell with omidubicel. This underscores the crucial need for additional research and development to broaden the spectrum of treatment options for this complex condition.
Detailed market assessment will be provided in the final report
Key Findings
As per DelveInsight's estimates, the potential drugs that can mark a significant change in the forecast period include xx, xx, and others.
This section focuses on the uptake rate of potential drugs expected to be launched in the market during 2020-2034. A medium uptake is expected by REGN7257.
Further detailed analysis of therapies drug uptake will be provided in the final report …
Aplastic Anemia Pipeline Development Activities
The report provides insights into different therapeutic candidates in Phase III and Phase II. It also analyzes key players involved in developing targeted therapeutics.
Pipeline Development Activities
The report covers information on collaborations, acquisitions and mergers, licensing, and patent details for aplastic anemia emerging therapy.
KOL Views
To keep up with current market trends, we take KOLs and SMEs' opinions working in the domain through primary research to fill the data gaps and validate our secondary research. Industry experts contacted for insights on aplastic anemia evolving treatment landscape, patient reliance on conventional therapies, patient's therapy switching acceptability, and drug uptake, along with challenges related to accessibility, include Medical/scientific writers; Professors; MD, FACS, Head of the Departments, Universities, and others.
Delveinsight's analysts connected with 30+ KOLs to gather insights; however, interviews were conducted with 10+ KOLs in the 7MM. Centers such as the East Tennessee State University, Dusseldorf Clinics Association, Duke University School of Medicine, etc. were contacted. Their opinion helps understand and validate current and emerging therapy treatment patterns or aplastic anemia market trends. This will support the clients in potential upcoming novel treatments by identifying the overall scenario of the market and the unmet needs.
Qualitative Analysis
We perform Qualitative and market Intelligence analysis using various approaches, such as SWOT analysis and Analyst views. In the SWOT analysis, strengths, weaknesses, opportunities, and threats in terms of disease diagnosis, patient awareness, patient burden, competitive landscape, cost-effectiveness, and geographical accessibility of therapies are provided. These pointers are based on the Analyst's discretion and assessment of the patient burden, cost analysis, and existing and evolving treatment landscape.
The analyst analyzes multiple emerging therapies based on relevant attributes such as safety, efficacy, frequency of administration, route of administration, and order of entry.
In efficacy, the trial's primary and secondary outcome measures are evaluated. Further, the therapies' safety is evaluated wherein the acceptability, tolerability, and adverse events are majorly observed, and it sets a clear understanding of the side effects posed by the drug in the trials.
Market Access and Reimbursement
Reimbursement is a crucial factor affecting the drug's market access. Often, the decision to reimburse comes down to the price of the drug relative to the benefit it produces in treated patients. To reduce the healthcare burden of these high-cost therapies, payers and other industry insiders are considering many payment models.
Patients prescribed PROMCATA might be eligible to receive a free 14-day supply by mail while beginning therapy. Additionally, Novartis also provides eligible patients with a temporary supply of PROMACTA through its Voucher Program. Through its Universal Copay Card, eligible patients with private insurance may pay USD 0 per month, with Novartis paying the remaining copay up to USD 15,000 per calendar year. Patients facing financial hardship and/or having no third-party insurance coverage might be eligible to receive PROMACTA for free through the Novartis Patient Assistance Foundation. About 99% of the patients being prescribed PROMACTA have it included in their health coverage, with more than 70% of Medicare and commercially insured patients paying less than USD 10,000 in out-of-pocket costs.
The report further provides detailed insights on the country-wise accessibility and reimbursement scenarios, cost-effectiveness scenario of approved therapies, programs making accessibility easier and out-of-pocket costs more affordable, insights on patients insured under federal or state government prescription drug programs, etc.
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