RARE DISEASE
Bullous pemphigoid
Bullous pemphigoid
Bullous pemphigoid
Drug discovery
8
drugs
With orphan designations
Overview
Bullous Pemphigoid (BP) is a chronic autoimmune blistering disease characterized by IgG/IgE autoantibodies targeting hemidesmosomal proteins BP180/BP230, leading to subepidermal separation, pruritus, and tense blisters. Diagnosis combines clinical presentation (urticarial plaques, flexural blisters), histopathology (eosinophil-rich subepidermal cleavage), and immunofluorescence (linear basement membrane IgG/C3 deposits). Treatment centers on corticosteroids and immunosuppressants, with emerging biologics targeting IL-4/IL-13, IgE, and complement pathways. Mortality rates remain elevated due to comorbidities and treatment-related complications [1][5][6].
Population
Primarily affects adults >70 years (median age 66–83), with incidence rising to 190–312 cases/million/year in those >80; rarely occurs <50 [2][12][14].
Incidence has increased 1.9- to 4.3-fold since 2000, linked to aging populations, neurocognitive disorders (e.g., dementia), and drug triggers (e.g., DPP-4 inhibitors) [2][5][10].
Burden
Mortality: 23% 1-year mortality, rising to 27–72% in frail elderly, driven by infections, thromboembolism, and comorbidities (stroke, diabetes) [4][15][17].
Quality of life: Severe pruritus, pain, and depression/anxiety rates of 31–38%; prolonged hospitalizations (+2.6 days vs. controls) [4][7][16].
Economic impact: High healthcare utilization (mean 7.3 hospital days) and costs from long-term immunosuppression [4][12][19].
Therapies
First-line: High-potency topical corticosteroids (e.g., clobetasol) or oral prednisone (0.5 mg/kg/day), tapered with steroid-sparing agents (doxycycline, methotrexate, azathioprine) [1][8][18].
Refractory cases: Biologics (rituximab, omalizumab) and targeted therapies (dupilumab, bertilimumab) show promise in clinical trials [3][13][17].
Adjuncts: Wound care, infection prevention, and monitoring for steroid toxicity (osteoporosis, diabetes) [1][6][16].
Categories: rare skin diseases
Research Papers
1,220 drug discovery papers about Bullous pemphigoid, with 2 first-in-class and 1 next-in-class emerging drug candidates forecasted to outperform the average preclinical success rate. Recent publications:
1,220 drug discovery papers about Bullous pemphigoid, with 2 first-in-class and 1 next-in-class emerging drug candidates forecasted to outperform the average preclinical success rate. Recent publications:
2026-08-13 | Transforming the treatment of autoimmune skin diseases: a journey through biologic therapies.
Autoimmune blistering diseases and autoimmune connective tissue diseases with prominent skin involvement are increasingly managed with biologic and targeted therapies. We review the evidence supporting these approaches and their practical positioning. We performed a PubMed/MEDLINE literature review (inception to February 2026) on biologic therapies for pemphigus, pemphigoid spectrum disorders, LABD, EBA, cutaneous lupus erythematosus, and dermatomyositis, including selected non-biologic small molecules, focusing on validated cutaneous outcomes. Evidence is strongest for B-cell depletion in pemphigus (including dose-optimization strategies) and for IL-4/IL-13 pathway blockade in BP, with additional data for anti-IgE therapy in selected BP phenotypes. In CLE, the most consistent cutaneous datasets involve pDC/type I interferon-axis modulation and BAFF inhibition, largely derived from systemic lupus cohorts; TYK2 inhibition has shown controlled benefit in CLE. In dermatomyositis, CDASI-based evidence is strongest for IVIg, with anti-IFNβ therapy and the TYK2/JAK1 inhibitor brepocitinib (positive in a phase 3 trial) showing cutaneous benefit; other biologics show limited trial signals. Biologic/targeted therapies should be positioned by disease phenotype and evidence strength, prioritizing steroid-sparing strategies in pemphigus and BP and using CLASI/CDASI-anchored outcomes to interpret lupus and dermatomyositis data. Evidence for rare AIBDs remains mostly case-level and should be framed accordingly.
2026-08-10 | Sitagliptin-associated Bullous Pemphigoid Complicated by Secondary Candidiasis, Urosepsis, and Gastrointestinal Bleeding in an Elderly Diabetic Woman.
Bullous pemphigoid (BP) is the most common autoimmune blistering disease in older adults and is increasingly associated with dipeptidyl peptidase-4 (DPP-4) inhibitors used in type 2 diabetes mellitus. Sitagliptin-associated BP is uncommon and may be complicated by secondary infections and multisystem involvement. A 74-year-old female with type 2 diabetes mellitus and hypertension presented with generalized weakness, burning micturition, abdominal pain, painful blistering skin lesions, and oral erosions. She had been receiving sitagliptin 100 mg once daily for approximately 2 years before symptom onset. Skin biopsy demonstrated a subepidermal bulla with eosinophilic infiltrates, suggestive of BP. Sitagliptin was discontinued after admission. The hospital course was complicated by bilateral pyelonephritis with multidrug-resistant urosepsis requiring bilateral double-J stenting, oral candidiasis caused by Candida albicans, recurrent gastrointestinal bleeding, and tubulovillous adenoma with high-grade dysplasia. Persistent dermatologic disease with markedly elevated immunoglobulin E levels necessitated omalizumab therapy, resulting in gradual improvement. Sitagliptin-associated BP should be considered in elderly diabetic patients presenting with blistering disorders after prolonged gliptin exposure. Early diagnosis, withdrawal of the suspected drug, histopathological confirmation, and multidisciplinary management are essential for favorable outcomes.
2026-08-02 | Bullous pemphigoid in patients who are frail: a case complicated by cerebral nocardiosis and Pneumocystis jirovecii pneumonia.
Bullous pemphigoid (BP) is the most common autoimmune blistering disorder in older patients and is typically managed with long-term immunosuppressive therapy tailored to the patient's comorbidities and overall health. Patients who are frail with concurrent haematological malignancies represent a particularly vulnerable population due to their impaired immunity and treatment-related immunosuppression. We report the case of an 85-year-old man with mantle cell lymphoma, previously treated with rituximab and ibrutinib, who developed BP confirmed by histology, direct immunofluorescence, indirect immunofluorescence and enzyme-linked immunosorbent assay. Initial disease control was achieved with systemic corticosteroids and methotrexate. Subsequently, he developed a subcutaneous thoracic nodule consistent with panniculitis, followed by cerebral nocardiosis and Pneumocystis jirovecii pneumonia. Despite successful remission of BP, his clinical course was complicated by life-threatening infections requiring prolonged hospitalization. This case highlights the delicate balance between controlling autoimmune disease and preventing severe opportunistic infections in older, frail patients with haematological malignancies. Current BP guidelines should be refined by integrating better frailty assessment, infection prophylaxis and tailored immunosuppressive strategies.
2026-08-02 | Factors associated with prolonged hospital stay in patients with autoimmune bullous diseases treated at an in-patient dermatology service.
Bullous diseases carry a great burden and are often linked to high healthcare costs. Patients with -bullous diseases are more vulnerable to infections and organ damage due to an incompetent skin barrier. To determine the demographic, clinical and laboratory factors associated with prolonged hospital stay in patients with autoimmune bullous diseases. This was a cross-sectional study, with a follow-up cohort, including all dermatology consultations in a university hospital between 2017 and 2022 to identify patients with autoimmune bullous disease. Duration of stay and other variables were obtained from the patients' medical records. A total of 83 patients were included in the study, of whom the majority had bullous pemphigoid (n = 45; 54%). There was significant metabolic comorbidity with hypertension (n = 51; 61%), diabetes (n = 31; 37%) and dyslipidaemia (n = 31; 37%). Fifty-eight per cent of patients had a prolonged hospital stay (>10 days). Among the associated variables, mucosal involvement, eosinophilia, in-hospital antibiotics and the absence of dipetidyl peptidase-4 (DPP4) inhibitor use stood out. Most patients with bullous diseases have prolonged hospital stays. The evaluation of mucous membranes, blood count and a basic chemical panel can help identify patients at greater risk of prolonged hospital stays. In addition, the use of DPP4 inhibitors and their rapid discontinuation could affect this outcome.
2026-07-29 | Treatment patterns for bullous pemphigoid in older adults: A population-based study.
Bullous pemphigoid (BP) is a chronic autoimmune blistering disease that disproportionately affects older adults and is associated with substantial morbidity, mortality, and treatment-related risks. Despite an expanding therapeutic landscape, real-world prescribing patterns in this population remain poorly characterized. To evaluate medication prescribing patterns among older adults with BP and assess the feasibility of using national claims data to characterize treatment exposures relevant to drug safety and risk stratification. We conducted a retrospective cohort study using Medicare fee-for-service claims data (2016-2020). Adults aged 65 years or older with BP were identified using ICD-10 codes. Prescription drug event data were linked to outpatient claims to capture medication use. Treatments were categorized into topical corticosteroids (by potency), oral immunomodulators and antibiotics, and biologic therapies. Descriptive analyses were performed to evaluate prescribing distributions. Among 10,959 patients with BP and 78,468 outpatient claims, we identified 19,663 prescriptions. Oral immunomodulators and antibiotics comprised 67.5% of prescriptions, followed by topical corticosteroids (32.5%) and biologics (0.04%). Prednisone accounted for 62.9% of oral prescriptions, followed by doxycycline (20.2%) and mycophenolate (6.0%). Among topical steroids, medium-potency agents were most common (40.6%), followed by super high-potency (38.2%). Biologic use was rare, with dupilumab accounting for most prescriptions. Systemic corticosteroids remain the predominant therapy for BP in older adults, with substantial variability in topical steroid use and minimal uptake of biologics. These findings highlight gaps in treatment optimization and support the need for risk-informed, steroid-sparing strategies in this high-risk population.
2026-08-13 | Transforming the treatment of autoimmune skin diseases: a journey through biologic therapies.
Autoimmune blistering diseases and autoimmune connective tissue diseases with prominent skin involvement are increasingly managed with biologic and targeted therapies. We review the evidence supporting these approaches and their practical positioning. We performed a PubMed/MEDLINE literature review (inception to February 2026) on biologic therapies for pemphigus, pemphigoid spectrum disorders, LABD, EBA, cutaneous lupus erythematosus, and dermatomyositis, including selected non-biologic small molecules, focusing on validated cutaneous outcomes. Evidence is strongest for B-cell depletion in pemphigus (including dose-optimization strategies) and for IL-4/IL-13 pathway blockade in BP, with additional data for anti-IgE therapy in selected BP phenotypes. In CLE, the most consistent cutaneous datasets involve pDC/type I interferon-axis modulation and BAFF inhibition, largely derived from systemic lupus cohorts; TYK2 inhibition has shown controlled benefit in CLE. In dermatomyositis, CDASI-based evidence is strongest for IVIg, with anti-IFNβ therapy and the TYK2/JAK1 inhibitor brepocitinib (positive in a phase 3 trial) showing cutaneous benefit; other biologics show limited trial signals. Biologic/targeted therapies should be positioned by disease phenotype and evidence strength, prioritizing steroid-sparing strategies in pemphigus and BP and using CLASI/CDASI-anchored outcomes to interpret lupus and dermatomyositis data. Evidence for rare AIBDs remains mostly case-level and should be framed accordingly.
2026-08-10 | Sitagliptin-associated Bullous Pemphigoid Complicated by Secondary Candidiasis, Urosepsis, and Gastrointestinal Bleeding in an Elderly Diabetic Woman.
Bullous pemphigoid (BP) is the most common autoimmune blistering disease in older adults and is increasingly associated with dipeptidyl peptidase-4 (DPP-4) inhibitors used in type 2 diabetes mellitus. Sitagliptin-associated BP is uncommon and may be complicated by secondary infections and multisystem involvement. A 74-year-old female with type 2 diabetes mellitus and hypertension presented with generalized weakness, burning micturition, abdominal pain, painful blistering skin lesions, and oral erosions. She had been receiving sitagliptin 100 mg once daily for approximately 2 years before symptom onset. Skin biopsy demonstrated a subepidermal bulla with eosinophilic infiltrates, suggestive of BP. Sitagliptin was discontinued after admission. The hospital course was complicated by bilateral pyelonephritis with multidrug-resistant urosepsis requiring bilateral double-J stenting, oral candidiasis caused by Candida albicans, recurrent gastrointestinal bleeding, and tubulovillous adenoma with high-grade dysplasia. Persistent dermatologic disease with markedly elevated immunoglobulin E levels necessitated omalizumab therapy, resulting in gradual improvement. Sitagliptin-associated BP should be considered in elderly diabetic patients presenting with blistering disorders after prolonged gliptin exposure. Early diagnosis, withdrawal of the suspected drug, histopathological confirmation, and multidisciplinary management are essential for favorable outcomes.
2026-08-02 | Bullous pemphigoid in patients who are frail: a case complicated by cerebral nocardiosis and Pneumocystis jirovecii pneumonia.
Bullous pemphigoid (BP) is the most common autoimmune blistering disorder in older patients and is typically managed with long-term immunosuppressive therapy tailored to the patient's comorbidities and overall health. Patients who are frail with concurrent haematological malignancies represent a particularly vulnerable population due to their impaired immunity and treatment-related immunosuppression. We report the case of an 85-year-old man with mantle cell lymphoma, previously treated with rituximab and ibrutinib, who developed BP confirmed by histology, direct immunofluorescence, indirect immunofluorescence and enzyme-linked immunosorbent assay. Initial disease control was achieved with systemic corticosteroids and methotrexate. Subsequently, he developed a subcutaneous thoracic nodule consistent with panniculitis, followed by cerebral nocardiosis and Pneumocystis jirovecii pneumonia. Despite successful remission of BP, his clinical course was complicated by life-threatening infections requiring prolonged hospitalization. This case highlights the delicate balance between controlling autoimmune disease and preventing severe opportunistic infections in older, frail patients with haematological malignancies. Current BP guidelines should be refined by integrating better frailty assessment, infection prophylaxis and tailored immunosuppressive strategies.
2026-08-02 | Factors associated with prolonged hospital stay in patients with autoimmune bullous diseases treated at an in-patient dermatology service.
Bullous diseases carry a great burden and are often linked to high healthcare costs. Patients with -bullous diseases are more vulnerable to infections and organ damage due to an incompetent skin barrier. To determine the demographic, clinical and laboratory factors associated with prolonged hospital stay in patients with autoimmune bullous diseases. This was a cross-sectional study, with a follow-up cohort, including all dermatology consultations in a university hospital between 2017 and 2022 to identify patients with autoimmune bullous disease. Duration of stay and other variables were obtained from the patients' medical records. A total of 83 patients were included in the study, of whom the majority had bullous pemphigoid (n = 45; 54%). There was significant metabolic comorbidity with hypertension (n = 51; 61%), diabetes (n = 31; 37%) and dyslipidaemia (n = 31; 37%). Fifty-eight per cent of patients had a prolonged hospital stay (>10 days). Among the associated variables, mucosal involvement, eosinophilia, in-hospital antibiotics and the absence of dipetidyl peptidase-4 (DPP4) inhibitor use stood out. Most patients with bullous diseases have prolonged hospital stays. The evaluation of mucous membranes, blood count and a basic chemical panel can help identify patients at greater risk of prolonged hospital stays. In addition, the use of DPP4 inhibitors and their rapid discontinuation could affect this outcome.
2026-07-29 | Treatment patterns for bullous pemphigoid in older adults: A population-based study.
Bullous pemphigoid (BP) is a chronic autoimmune blistering disease that disproportionately affects older adults and is associated with substantial morbidity, mortality, and treatment-related risks. Despite an expanding therapeutic landscape, real-world prescribing patterns in this population remain poorly characterized. To evaluate medication prescribing patterns among older adults with BP and assess the feasibility of using national claims data to characterize treatment exposures relevant to drug safety and risk stratification. We conducted a retrospective cohort study using Medicare fee-for-service claims data (2016-2020). Adults aged 65 years or older with BP were identified using ICD-10 codes. Prescription drug event data were linked to outpatient claims to capture medication use. Treatments were categorized into topical corticosteroids (by potency), oral immunomodulators and antibiotics, and biologic therapies. Descriptive analyses were performed to evaluate prescribing distributions. Among 10,959 patients with BP and 78,468 outpatient claims, we identified 19,663 prescriptions. Oral immunomodulators and antibiotics comprised 67.5% of prescriptions, followed by topical corticosteroids (32.5%) and biologics (0.04%). Prednisone accounted for 62.9% of oral prescriptions, followed by doxycycline (20.2%) and mycophenolate (6.0%). Among topical steroids, medium-potency agents were most common (40.6%), followed by super high-potency (38.2%). Biologic use was rare, with dupilumab accounting for most prescriptions. Systemic corticosteroids remain the predominant therapy for BP in older adults, with substantial variability in topical steroid use and minimal uptake of biologics. These findings highlight gaps in treatment optimization and support the need for risk-informed, steroid-sparing strategies in this high-risk population.
Access all drug discovery papers and probability of success in trials forecasts:
Access all drug discovery papers and probability of success in trials forecasts:
Drug Discovery Landscape
8 orphan drug designations for Bullous pemphigoid, including 1 approved therapy.
8 orphan drug designations for Bullous pemphigoid, including 1 approved therapy.
Drug | Therapy type | Regulator | Orphan designation | Approval | Sponsor |
|---|---|---|---|---|---|
rituximab | antibodies | FDA | 2025-10-16 | — | ODDIFACT SAS |
Nomacopan | proteins | EMA | 2020-06-26 | — | Akari Malta Limited |
nomacopan | combination | FDA | 2019-09-12 | — | Akari Therapeutics Plc |
dupilumab [Dupixent] | antibodies | FDA | 2019-08-21 | 2025-06-18 | Regeneron Pharmaceuticals, Inc. |
Bertilimumab | antibodies | EMA | 2018-08-24 | — | IQVIA RDS Ireland Limited |
bertilimumab | antibodies | FDA | 2018-08-17 | — | Alexion Pharmaceuticals, Inc. |
humanized IgG4 Monoclonal Antibody against total complement component 1, subcomponent s (C1s) | antibodies | FDA | 2017-08-17 | — | Recordati Rare Diseases Inc. |
Dimethyl fumarate | small molecules | EMA | 2016-07-14 | — | Novalis Investments S.L |
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