주요 7개국 온식 자가면역성 용혈성 빈혈(WAIHA)의 전체 시장 규모는 2023년에 3억 달러에 달했습니다. WAIHA는 자가면역성 용혈성 빈혈의 가장 일반적인 유형이며, 성인 증례의 약 70-80%, 소아 증례의 50%를 차지하고 있습니다. 온식 AIHA 치료에는 주로 코르티코스테로이드가 이용되며, 기본적인 어프로치로서 널리 이용되고 있습니다. 부신피질 스테로이드가 효과가 없는 것으로 판명되면 비장절제술을 시행할 가능성도 있습니다. 리툭시맙은 현재 기존 치료에 저항성이 있는 환자들에게 효과적인 대안으로 여겨지고 있으며, 면역억제제의 사용은 특히 만성 중증 난치성 환자들에게 효과적입니다.
또한 아자티오프린, 사이클로포스파미드, 모페틸 미코페놀산과 같은 면역억제제는 특히 만성 중증 난치성 AIHA 환자에서 중요한 보조 약물로 등장하고 있습니다.
난치성 또는 재발 사례의 경우 연속적인 치료 라인이 사용되며, AIHA는 여전히 치료하기 어려운 질병이며, AIHA의 신약 연구개발이 중요한 이유입니다.
wAIHA의 근본적인 병태생리를 다루는 혁신적인 표적 치료가 필요하며, 유망한 새로운 치료법으로는 새로운 B세포/형질세포 표적 치료제, 보체 억제제, 신생아 Fc 수용체 차단별 병원성 자가항체 제거제 등이 있습니다.
Obexelimab, Ianalumab, Nipocalimab, Rilzabrutinib, Povetacicept와 같은 새로운 약물은 치료 옵션을 늘리고 리툭시맙 후 재발성/리툭시맙 내성 wAIHA의 공백을 메울 수 있습니다. 또한 이러한 새로운 표적치료제는 초급성기 미충족 수요를 충족시킬 수 있는 수단이 될 수 있습니다.
주요 7개국의 온식 자가면역성 용혈성 빈혈(WAIHA) 시장에 대해 조사했으며, 시장의 개요와 역학, 환자 동향, 새로운 치료법, 2034년까지의 시장 규모 예측 및 의료 미충족 요구 등을 제공하고 있습니다.
DelveInsight's "Warm Autoimmune Hemolytic Anemia (wAIHA) - Market Insights, Epidemiology, and Market Forecast - 2034" report delivers an in-depth understanding of wAIHA, historical and forecasted epidemiology as well as the wAIHA market trends in the United States, EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan.
The wAIHA market report provides current treatment practices, emerging drugs, wAIHA market share of individual therapies, and current and forecasted wAIHA market size from 2020 to 2034, segmented by seven major markets. The report also covers current wAIHA treatment practices/algorithms and unmet medical needs to curate the best of the opportunities and assess the underlying potential of the market.
Study Period: 2020-2034
wAIHA Overview
Warm autoimmune hemolytic anemia (wAIHA) is a type of autoimmune disorder characterized by the destruction of red blood cells (hemolysis) due to the body's immune system mistakenly targeting its red blood cells. In wAIHA, the antibodies produced by the immune system bind to red blood cells at body temperature (hence "warm") and mark them for destruction by the spleen and other organs. This leads to a decrease in the number of circulating red blood cells, resulting in anemia. Symptoms of wAIHA can vary but often include fatigue, weakness, pallor, shortness of breath, and jaundice (yellowing of the skin and eyes). The condition can be primary (idiopathic) or secondary to underlying conditions such as autoimmune diseases, infections, lymphoproliferative disorders, or certain medications. Diagnosis involves blood tests, and treatment typically includes immunosuppressive medications to suppress the abnormal immune response and alleviate symptoms.
wAIHA Diagnosis
Diagnosing wAIHA involves several steps to confirm the presence of hemolysis (destruction of red blood cells) and to identify the underlying cause. Initially, a complete blood count (CBC) is conducted to assess hemoglobin levels, red blood cell count, and other parameters indicative of anemia. Peripheral blood smear examination may reveal signs of hemolysis, such as red blood cell destruction and the presence of spherocytes (abnormally shaped red blood cells). Direct antiglobulin test (DAT), also known as the Coombs test, is performed to detect antibodies or complement proteins bound to the surface of red blood cells. A positive DAT result confirms the presence of autoimmune-mediated hemolysis. Further tests, including serum protein electrophoresis, autoimmune serology, and bone marrow examination, may be conducted to identify underlying conditions associated with wAIHA, such as autoimmune diseases, infections, or malignancies. Overall, a combination of clinical findings and laboratory tests is crucial for the accurate diagnosis of wAIHA and for determining appropriate treatment strategies.
Further details related to diagnosis will be provided in the report...
wAIHA Treatment
The treatment of warm autoimmune hemolytic anemia (wAIHA) aims to suppress the autoimmune response, alleviate symptoms, and manage complications. Corticosteroids, such as prednisone, are often the first-line therapy, as they help suppress the immune system's abnormal activity and reduce red blood cell destruction. In cases where corticosteroids are ineffective or poorly tolerated, other immunosuppressive medications may be used, including azathioprine, rituximab, mycophenolate mofetil, or cyclophosphamide. Intravenous immunoglobulin (IVIG) therapy may also be considered for rapid symptom relief in severe cases. In some instances, splenectomy (surgical removal of the spleen) may be recommended, particularly if other treatments fail or are contraindicated. However, splenectomy carries risks and is generally reserved for refractory cases. Additionally, supportive care measures, such as blood transfusions to alleviate severe anemia and folic acid supplementation to support red blood cell production, may be necessary. Long-term management involves monitoring for disease activity, adjusting treatment as needed, and addressing any underlying conditions contributing to wAIHA. Collaboration with hematologists and rheumatologists is essential to develop individualized treatment plans and optimize outcomes for patients with wAIHA.
Further details related to treatment will be provided in the report.....
The wAIHA epidemiology chapter in the report provides historical as well as forecasted epidemiology segmented by the total diagnosed prevalent cases of autoimmune hemolytic anemia, total prevalent cases of wAIHA, type-specific cases of wAIHA, gender-specific cases of wAIHA, and age-specific cases of wAIHA in the 7MM market covering the United States, EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan from 2020 to 2034.
The drug chapter segment of the wAIHA report encloses a detailed analysis of the late-stage (Phase III and Phase II/III) and early-stage (Phase I/II) pipeline drugs. The current key players for emerging drugs and their respective drug candidates include Zenas BioPharma (Obexelimab) and Novartis/MorphoSys (Ianalumab). The drug chapter also helps understand the wAIHA clinical trial details, expressive pharmacological action, agreements and collaborations, approval, and patent details, and the latest news and press releases.
Emerging Drugs
Obexelimab (ZB012): Zenas BioPharma
Obexelimab is a novel bifunctional antibody with first-in-class potential that inhibits B-cell lineages that express CD19. Simultaneous binding to CD19 and Fc?RIIB by obexelimab mimics a naturalantigen-antibody complex and downregulates B-cell activity. In early-stage clinical studies, obexelimab effectively demonstrated inhibition of B-cell function without depleting the cells and generated an encouraging treatment effect in patients with multiple autoimmune diseases. Currently, the drug is being evaluated in Phase III (SApHiAre trial) clinical stage of development.
In September 2023, Zenas BioPharma entered into a license and collaboration agreement with Bristol Myers Squibb to develop and commercialize obexelimab for autoimmune diseases in Japan, South Korea, Taiwan, Singapore, Hong Kong, and Australia.
Nipocalimab (M281): Johnson & Johnson Innovative Medicine
Nipocalimab is a fully human monoclonal antibody that targets the neonatal crystallizable fragment receptor (FcRn) with potential immunomodulating activity. Upon administration, nipocalimab targets and binds to FcRn at the IgG binding site, thereby preventing the interaction between FcRn and the serum protein IgG. By preventing FcRn/IgG binding, nipocalimab blocks the FcRn-mediated rescue of IgG, enables IgG degradation, and prevents IgG-mediated inflammation. Nipocalimab was granted FTD for wAIHA in July 2019 and ODD in December 2019. Currently, the drug is being evaluated in Phase II/III (NCT04119050) clinical stage of development.
Drug Class Insight
BTK inhibitor
BTK plays a crucial role in B-cell receptor signaling, which is involved in the activation and proliferation of B cells. By inhibiting BTK, these medications can modulate B-cell function and the immune response, potentially dampening the autoimmune reaction responsible for wAIHA. Rilzabrutinib was an investigational drug, and there was limited information available about its use specifically for the treatment of warm autoimmune hemolytic anemia (wAIHA). However, given its mechanism of action as a Bruton's tyrosine kinase (BTK) inhibitor, similar to other drugs in this class like ibrutinib, there is potential for rilzabrutinib to be explored as a treatment option for wAIHA.
The most common therapy and the cornerstone of treatment for warm AIHA are corticosteroids. If these are ineffective, a splenectomy can be considered. Rituximab has become an option in refractory disease, and the use of immunosuppressors can be helpful in chronic severe refractory cases.
While glucocorticoids are considered the first-line treatment in WAIHA, this was empirically derived. Mechanisms of actions include suppression of autoantibody production, reduction in autoantibody affinity, and decreased destruction of erythrocytes by splenic macrophages, perhaps by diminished expression of Fc? receptors. Rituximab is used in diagnosed severe cases or in cases where long-term corticosteroids should be avoided. Although rituximab (an anti-CD20 antibody) is considered a second-line treatment, the combination of rituximab and prednisone at relatively low doses (100 mg once weekly, four times) is increasingly becoming a first-line treatment. With the advent of rituximab, azathioprine, cyclophosphamide, cyclosporine, and intravenous immunoglobulin became second or third-line treatments.
Conventional immunosuppressive drugs (such as azathioprine, cyclophosphamide, and cyclosporine), although widely used in clinical practice mainly as steroid-sparing agents, are moving to later lines.
Rilzabrutinib, a reversible, covalent Bruton tyrosine kinase (BTK) inhibitor, is also being studied in a Phase IIb trial. This drug also inhibits phagocytosis via interaction with the syk pathway. Several other therapies, including Obexelimab (ZB012), Ianalumab, and Povetacicept, are expected to fill the treatment gap and fulfill the current unmet needs
Key Findings
This section focuses on the uptake rate of potential drugs expected to be launched in the market during 2024-2034. The landscape of wAIHA treatment has experienced a transformation with the uptake of novel drugs. These innovative therapies are redefining standards of care. Furthermore, the increased uptake of these transformative drugs is a testament to the unwavering dedication of hematologists, Researchers, professionals, and the entire healthcare community in their tireless pursuit of advancing eye care. This momentous shift in treatment paradigms is a testament to the power of research, collaboration, and human resilience.
Warm Autoimmune Hemolytic Anemia (wAIHA) Pipeline Development Activities
The report provides insights into therapeutic candidates in Phase III, and Phase II. It also analyzes key players involved in developing targeted therapeutics. Companies like Zenas BioPharma and Novartis/MorphoSys actively engage in late-stage research and development efforts for wAIHA. The pipeline of wAIHApossesses a few potential drugs. However, there is a positive outlook for the therapeutics market, with expectations of growth during the forecast period (2024-2034).
Pipeline Development Activities
The report covers information on collaborations, acquisitions and mergers, licensing, and patent details for wAIHA 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 the wAIHA evolving treatment landscape, patient reliance on conventional therapies, patient therapy switching acceptability, and drug uptake, along with challenges related to accessibility, including Hematologist, Rheumatologist, Hematology-Oncology Specialist, and others.
DelveInsight's analysts connected with 15+ KOLs to gather insights; however, interviews were conducted with 5+ KOLs in the 7MM. Centers such as the University of California Los Angeles Medical Center, Professor of Massachusetts General Hospital, MD, Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS), etc., were contacted. Their opinion helps understand and validate current and emerging therapy treatment patterns or wAIHA 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.
Market Access and Reimbursement
wAIHA is characterized by evidence of red blood cell (RBC) hemolysis and a direct antiglobulin test positive for IgG and sometimes complement. While varying with the extent of the compensatory increase in RBC production, symptoms of anemia predominate, as does jaundice, the latter often exacerbated by concurrent Gilbert's syndrome. Initial treatment with corticosteroids is highly effective, with over 85% of patients responding but with less than one-third maintaining that response upon weaning. Subsequent rituximab administration in those failing corticosteroids provides complete remission in over 75% of patients and may be long-lasting. Over 50% of patients failing rituximab respond to erythropoiesis-stimulating agents or immunosuppressive agents. Splenectomy is best deferred if possible but does offer long-term remission in over two-thirds of patients. Current treatment approaches include corticosteroids, rituximab, immunosuppressive drugs, and splenectomy, but there are, so far, no disease-targeted therapies for wAIHA.
Rituximab: NICE
Rituximab is not licensed for treating autoimmune hemolytic anemia, so use for this indication is off-label. Rituximab 10 mg/mL concentrate for solution for intravenous infusion.
Comparing the cost of rituximab with other therapies for autoimmune hemolytic anemia is difficult because there is a lack of evidence to confirm the optimal dose, guide the use of recurrent courses in refractory cases, and confirm the advice on other aspects of the clinical pathway, such as combination with other treatments.
ICD-10 code D59.11 for wAIHA is a medical classification as listed by WHO under the range of diseases of the blood and blood-forming organs and certain disorders involving the immune mechanism.
Detailed market access and reimbursement assessment will be provided in the final report.
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