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市場調査レポート
商品コード
1605440
尋常性天疱瘡(PV)市場 - 市場の洞察、疫学、市場予測:2034年Pemphigus Vulgaris (PV) - Market Insight, Epidemiology And Market Forecast - 2034 |
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尋常性天疱瘡(PV)市場 - 市場の洞察、疫学、市場予測:2034年 |
出版日: 2024年11月01日
発行: DelveInsight
ページ情報: 英文 97 Pages
納期: 2~10営業日
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尋常性天疱瘡(PV)は、口や喉、時には生殖器などの皮膚や粘膜に痛みを伴う水疱やびらんを認める重篤で稀な自己免疫疾患です。この疾患は、表皮の細胞接着に不可欠なタンパク質であるデスモグレインを免疫系が誤って攻撃することにより発症し、細胞の剥離と水疱形成を引き起こします。
天疱瘡に伴う水疱は一般的に弛緩性で壊れやすく、主に頭皮、顔面、胸部、背部などの部位、特に口などの粘膜に形成されます。容易に破裂し、しばしば痛みを伴う開放性のただれを生じ、治癒に時間がかかります。より深い皮膚層を侵し、より強固で弾力性のある水疱を生じる天疱瘡とは異なり、天疱瘡はより表在性の皮膚層を侵し、繊細で容易に破裂する水疱を生じます。
尋常性天疱瘡の診断には、生検および抗体検査、特に抗デスモグレイン抗体の同定による、水疱性類天疱瘡や他のタイプの天疱瘡などの他の水疱形成性疾患との鑑別を行います。
治療は、主に副腎皮質ステロイド、アザチオプリンやミコフェノラートなどの免疫抑制剤、自己抗体産生を担うB細胞を標的とするリツキシマブ別生物学的療法によって、水疱形成を引き起こす自己免疫活性を制御することに重点を置いています。現在、リツキサン(リツキシマブ)は尋常性天疱瘡の治療で唯一FDAに承認されている治療法であり、特に中等度から重度の症例で寛解を達成するための効果的な選択肢となっており、多くの場合、コルチコステロイドの投与量を少なくすることが可能です。
その他の支持療法としては、感染予防のための抗生物質投与、口腔内病変の疼痛管理、治癒を助けるための適切な創傷ケアなどがあります。再発の傾向があるにもかかわらず、綿密なモニタリングとこれらの治療により、多くの患者がPVを管理することができます。
主要7ヶ国における尋常性天疱瘡の市場規模では、米国が最も高い市場シェア、すなわち2023年には75%以上を占め、次いでドイツが続きます。EU4ヶ国と英国の中では、ドイツが2023年の市場規模のほぼ25%を占めています。米国は2023年に約2億5,000万米ドルでした。利用可能な治療法の中で、リツキシマブはPV市場を一変させた薬剤とみられています。
当レポートでは、主要7ヶ国における尋常性天疱瘡(PV)市場について調査し、市場の概要とともに、疫学、患者動向、新たな治療法、2034年までの市場規模予測、および医療のアンメットニーズなどを提供しています。
Report Summary
Market
A few key players are leading the treatment landscape of Pemphigus Vulgaris, such as Roche, Cabaletta Bio, and others. The details of the country-wise and therapy-wise market size have been provided below.
The section dedicated to drugs in the Pemphigus Vulgaris report provides an in-depth evaluation of late-stage pipeline drugs (Phase III and Phase II) related to Pemphigus Vulgaris. The drug chapters section provides valuable information on various aspects related to clinical trials of Pemphigus Vulgaris, such as the pharmacological mechanisms of the drugs involved, designations, approval status, patent information, and a comprehensive analysis of the pros and cons associated with each drug. Furthermore, it presents the most recent news updates and press releases on drugs targeting Pemphigus Vulgaris.
Marketed Therapies
RITUXAN (rituximab): Roche
RITUXAN (rituximab) is the only FDA-approved therapy for the treatment of pemphigus vulgaris. It serves as a key treatment, especially for moderate to severe cases. As the first biologic therapy approved by the FDA for this condition, RITUXAN represents a significant advancement in over 60 years. The FDA has granted RITUXAN several important designations, including Priority Review, Breakthrough Therapy Designation, and Orphan Drug Designation, emphasizing its critical role in treating PV. Rituximab is a CD20-directed cytolytic antibody that targets B-cells, which are responsible for producing the autoantibodies that attack the skin and mucous membranes in PV. By depleting these B-cells, RITUXAN reduces the production of harmful antibodies, leading to improved symptoms and better disease control.
Emerging Therapies
CABA-201 (4-1BB CD19-CAR T): Cabaletta Bio
CABA-201 is designed to deeply and transiently deplete CD19-positive B cells following a one-time infusion, which may enable an "immune system reset" with the potential for durable remission of therapy in patients with autoimmune diseases. To date, Cabaletta has received clearance from the FDA for Investigational New Drug (IND) applications for CABA-201 in multiple autoimmune conditions, including Systemic Lupus Erythematosus (SLE), myositis, systemic sclerosis and generalized Myasthenia Gravis (gMG). Cabaletta is conducting four RESET Phase I/II clinical trials with a total of nine cohorts that can advance simultaneously, employing a similar parallel cohort design and starting dose of 1 x 106 cells/kg without a dose escalation requirement. In January 2020, Cabaletta Bio announced that the US Food and Drug Administration (FDA) had granted an Orphan Drug Designation for the Company's lead product candidate, DSG3-CAART, for the treatment of pemphigus vulgaris.
Currently, RITUXAN (rituximab) is the only FDA-approved therapy for the treatment of pemphigus vulgaris (PV). It serves as a key treatment, especially for moderate to severe cases. As the first biologic therapy approved by the FDA for this condition, RITUXAN represents a significant advancement in over 60 years.
A CD19-CAR T-cell therapy is being developed by Cabaletta Bio for treating autoimmune diseases, including PV. Currently, in mid-stage development, CABA-201 is designed to deeply and transiently deplete CD19-positive B cells following a one-time infusion, potentially enabling an "immune system reset" and durable disease remission. Cabaletta has received FDA clearance for Investigational New Drug (IND) applications for CABA-201 in several autoimmune conditions, and the therapy is being tested in multiple RESET Phase I/II clinical trials.
In a nutshell, not many potential therapies are being investigated to manage Pemphigus Vulgaris. Even though it is too soon to comment on the above-mentioned promising candidate to enter the market during the forecast period (2024-2034). Eventually, this drug will create a significant difference in the landscape of PV in the coming years. The treatment space is expected to experience a significant positive shift in the coming years owing to the improvement in healthcare spending worldwide.
Pemphigus Vulgaris (PV) Disease Understanding and Treatment
Pemphigus Vulgaris (PV) Overview
Pemphigus is a rare autoimmune blistering disorder that affects the outermost layer of the skin (epidermis) and mucous membranes. It is characterized by the formation of fragile blisters and lesions that rupture easily, leaving painful sores. This condition arises when the immune system mistakenly produces autoantibodies-particularly against proteins called desmoglein 1 and desmoglein 3, which are essential for maintaining the adhesion between skin cells. The disruption of these cellular junctions leads to a loss of skin integrity, causing the skin cells to separate, a process known as acantholysis. As a result, fluid accumulates between the skin layers, leading to the formation of blisters.
Pemphigus Vulgaris (PV) Diagnosis
The current diagnosis of PV involves a combination of clinical assessment, histopathological examination, and advanced immunological techniques such as direct and indirect immunofluorescence and ELISA. The diagnosis of PV should be suspected in any patient with mucocutaneous erosions or blisters. The oral mucosa is the first site of involvement in the majority of cases, and PV may remain confined to the mucosal surfaces or extend to involve the skin (average lag period of 4 months).
Pemphigus Vulgaris (PV) Treatment
The treatment of Pemphigus Vulgaris has significantly improved with the use of systemic corticosteroids, which reduced the disease's mortality rate by 60%. These steroids remain the first-line therapy, particularly for mild cases, though they require careful monitoring due to side effects like infections and osteoporosis. For moderate-to-severe PV, rituximab, an anti-CD20 monoclonal antibody, is commonly used in combination with corticosteroids, helping to deplete B-cells responsible for producing the autoantibodies that attack the skin. Steroid-sparing agents like azathioprine and mycophenolate mofetil (MMF) are also used to reduce steroid dependence and minimize side effects. For refractory cases, treatments such as IVIg and cyclophosphamide may be considered. While these therapies have improved outcomes, PV treatment remains complex, requiring ongoing management to balance efficacy and side effects.
The Pemphigus Vulgaris epidemiology chapter in the report provides historical as well as forecasted epidemiology segmented by total diagnosed prevalent cases, Gender-specific cases, Age-specific cases, Severity-specific cases, and total treated cases of Pemphigus Vulgaris in the United States, EU4 countries (Germany, France, Italy, Spain) and the United Kingdom, and Japan from 2020 to 2034.
KOL Views
To stay abreast of the latest trends in the market, we conduct primary research by seeking the opinions of Key Opinion Leaders (KOLs) and Subject Matter Experts (SMEs) who work in the relevant field. This helps us fill any gaps in data and validate our secondary research.
We have reached out to industry experts to gather insights on various aspects of Pemphigus Vulgaris, including the evolving treatment landscape, patients' reliance on conventional therapies, their acceptance of therapy switching, drug uptake, and challenges related to accessibility. The experts we contacted included medical/scientific writers, professors, and researchers from prestigious universities in the US, Europe, the UK, and Japan.
Our team of analysts at Delveinsight connected with more than 15 KOLs across the 7MM. We contacted institutions such as the Johns Hopkins University School of Medicine, Stanford University School of Medicine, University of California, etc., among others. By obtaining the opinions of these experts, we gained a better understanding of the current and emerging treatment patterns in the Pemphigus Vulgaris market, which will assist our clients in analyzing the overall epidemiology and market scenario.
We perform Qualitative and Market Intelligence analysis using various approaches, such as SWOT analysis and Conjoint Analysis. 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.
Conjoint Analysis analyzes multiple approved and emerging therapies based on relevant attributes such as safety, efficacy, frequency of administration, designation, route of administration, and order of entry. Scoring is given based on these parameters to analyze the effectiveness of therapy.
In efficacy, the trial's primary and secondary outcome measures are evaluated; for instance, in trials for Pemphigus Vulgaris, one of the most important primary endpoints was achieving hemolysis control, LDH normalization, etc. Based on these, the overall efficacy is 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. In addition, the scoring is also based on the route of administration, order of entry and designation, probability of success, and the addressable patient pool for each therapy. According to these parameters, the final weightage score and the ranking of the emerging therapies are decided.
Market Access and Reimbursement
Because newly authorized drugs are often expensive, some patients escape receiving proper treatment or use off-label, less expensive prescriptions. Reimbursement plays a critical role in how innovative treatments can enter the market. The cost of the medicine, compared to the benefit it provides to patients who are being treated, sometimes determines whether or not it will be reimbursed. Regulatory status, target population size, the setting of treatment, unmet needs, the number of incremental benefit claims, and prices can all affect market access and reimbursement possibilities.
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.