Anti-Glomerular Basement Membrane (Anti-GBM) Disease - Market Insights, Epidemiology, and Market Forecast - 2030

The ‘Anti-Glomerular Basement Membrane (Anti-GBM) Disease - Market Insights, Epidemiology, and Market Forecast-2030’ report delivers an in-depth understanding of the Anti-GBM disease, historical and forecasted epidemiology as well as the Anti-GBM Disease market trends in the United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan.

The Anti-GBM Disease market report provides current treatment practices, emerging drugs, Anti-GBM Disease market share of the individual therapies, and current and forecasted Anti-GBM Disease market size from 2018 to 2030 segmented by seven major markets. The Report also covers current Anti-GBM Disease treatment practice/algorithm, market drivers, market barriers, and unmet medical needs to curate the best of the opportunities and assesses the underlying potential of the market.

Geography Covered

• The United States
• EU5 (Germany, France, Italy, Spain, and the United Kingdom)
• Japan

Study Period: 2018-2030

Anti-GBM Disease: Disease Understanding and Treatment Algorithm

Anti-Glomerular Basement Membrane (Anti-GBM) Disease Overview

Anti-Glomerular Basement Membrane (anti-GBM) disease (also known as Goodpasture syndrome or disease) is a rare but serious autoimmune disease that causes inflammation in the kidneys and lungs. Anti-GBM disease may affect only the kidneys; however, when it causes both kidney and lung disease, it is called Goodpasture’s syndrome. In anti-GBM disease, the immune system mistakenly makes “anti-GBM antibodies” that attack the lungs and kidneys, leading to bleeding and inflammation in the organs. It is also characterized by the inflammation of the filtering structures (glomeruli) of the kidneys (glomerulonephritis) and excessive bleeding into the lungs (pulmonary hemorrhaging). Autoimmune syndromes occur when the body’s natural defense systems (antibodies) against invading or foreign organisms begin to attack the body’s tissue for unknown reasons.

Anti-GBM disease can affect just the kidneys or both the lungs and kidneys (it is uncommon for only the lungs to be affected). These organs are affected because the basement membrane that is targeted or attacked in this disease is only accessible to antibodies in the kidneys and lungs (not in other organs).

The anti-GBM is the most specific term and refers to the presence of renal and pulmonary involvement, along with anti-glomerular basement membrane antibodies. The spectrum of the disease may present classically or glomerulonephritis alone. In anti-GBM disease, the body creates autoantibodies that recognize and attach to the basement membrane, which is a part of the wall of capillary blood vessels in the kidneys and lungs. Once the autoantibodies attach to the basement membrane, this creates a signal to the body’s immune system to attack.

Anti-Glomerular Basement Membrane (Anti-GBM) Disease Diagnosis

Patients are tested for serum anti-GBM antibodies by indirect immunofluorescence testing or, when available, direct enzyme-linked immunosorbent assay (ELISA) with recombinant or human NC-1 alpha3. The presence of these antibodies confirms the diagnosis. Anti-neutrophil cytoplasmic antibodies (ANCA) testing is seen to be positive (in a peripheral pattern) only in 25% of patients with Goodpasture syndrome.

Anti-GBM antibodies can be tested with different diagnostic methods including Physical examination, anti-GBM antibody testing, Anti-neutrophil cytoplasmic antibody (ANCA) testing, renal biopsy, alveolar hemorrhage, and chest radiograph. In patients with evidence of diffuse alveolar hemorrhage and renal involvement, kidney biopsy is considered to identify the underlying cause and help direct therapy. Percutaneous kidney biopsy is the preferred invasive procedure to prove the diagnosis of anti-GBM disease. Renal biopsy provides a comparatively higher yield than lung biopsy, but transbronchial or open lung biopsy may be performed in cases where a renal biopsy cannot be performed. The biopsy tissue must also be processed for light microscopy, immunofluorescence, and electron microscopy.

Anti-Glomerular Basement Membrane (Anti-GBM) Disease Treatment

The main goal of the treatment of anti-GBM disease is to remove the harmful antibodies from the blood. Hospitalization is required for diagnosis and treatment, close monitoring, and supportive care in patients with anti-glomerular basement membrane (GBM) antibody disease. Patients may initially require intensive care.

Treatment requires a process called plasma exchange, which involves using a machine to remove anti-GBM antibodies from the bloodstream. This is often done daily for 2 weeks. In addition, immunosuppressive drugs such as steroids and cyclophosphamide, are used to suppress inflammation and stop further antibody production. Treatment usually continues for 6 months after the diagnosis is made.

Patients with moderate glomerulonephritis (serum creatinine level < 5 mg/dL and crescents in < 50-75% of glomeruli) and patients with the acute disease are likely to respond to therapy. The treatment can consist of repeated plasmapheresis combined with glucocorticoids and cyclophosphamide.

Patients with advanced disease (serum creatinine level >5 mg/dL and crescents in >75% of glomeruli) and histologic signs of chronicity are unlikely to improve with any therapy and should be spared the clinically significant risks of aggressive treatment. Supportive care and eventual renal transplantation are recommended. Patients who are ANCA positive with clinical presentation significant with vasculitis are likely to benefit from aggressive therapy independent of the severity of the disease. Most patients with pulmonary hemorrhage respond rapidly to methylprednisolone pulses, plasma exchange, or plasmapheresis. Patients with mild renal disease who do not have pulmonary hemorrhage may be successfully treated with prednisone alone.

Rituximab, a chimeric monoclonal antibody targeting the pan B-cell marker CD20, has been used as adjunctive or second-line therapy in resistant cases or when cyclophosphamide is contraindicated. At present, there is insufficient evidence to recommend it as first-line therapy for patients with anti-GBM antibody disease.

Anti-Glomerular Basement Membrane (Anti-GBM) Disease Epidemiology

The Anti-GBM Disease epidemiology division provides insights about historical and current Anti-GBM Disease patient pool and forecasted trends for every seven major countries. It helps to recognize the causes of current and forecasted trends by exploring numerous studies and views of key opinion leaders. This part of The report also provides the incident patient pool and their trends along with assumptions undertaken.

Key Findings

In the year 2020, the total incident cases of Anti-GBM Disease was 1,117 cases in the 7MM which are expected to grow during the study period, i.e., 2018-2030.

The disease epidemiology covered in the report provides historical as well as forecasted Anti-GBM epidemiology segmented as Total incident Cases of anti-GBM disease in the 7MM covering the United States, EU5 countries (Germany, France, Italy, Spain, and the United Kingdom), and Japan from 2018 to 2030.

Country Wise - Anti-Glomerular Basement Membrane (Anti-GBM) Disease Epidemiology

Estimates show that the highest cases of Anti-GBM Disease in the 7MM were in United States, followed by the Germany, France, the United Kingdom, Italy, Spain, and Japan in 2020.

• In the United States, the total number of incident cases of Anti-GBM Disease was 532 cases in the year 2020 which are expected to grow during the study period, i.e., 2018-2030.
• In the year 2020, the total incident cases of Anti-GBM Disease were 522 cases in EU-5 which are expected to grow during the study period, i.e., 2018-2030.
• In Japan, the total number of incident cases of Anti-GBM Disease was 63 cases in the year 2020 which are expected to grow during the study period, i.e., 2018-2030.

Anti-Glomerular Basement Membrane (Anti-GBM) Disease Drug Chapters

The drug chapter segment of the Anti-GBM Disease report encloses the detailed analysis of Anti-GBM Disease late stage pipeline drugs. It also helps to understand the Anti-GBM Disease clinical trial details, expressive pharmacological action, agreements and collaborations, approval and patent details, advantages and disadvantages of each included drug, and the latest news and press releases.

Anti-Glomerular Basement Membrane (Anti-GBM) Disease Emerging Drugs

Idefirix (Imlifidase/HMed-IdeS): Hansa Biopharma

Idefirix (Imlifidase/HMed-IdeS) is a unique intravenous antibody-cleaving enzyme originating from Streptococcus pyogenes. It specifically targets IgG and inhibits IgG-mediated immune response is being developed by Hansa Biopharma AB for treatment of transplant rejection and rare IgG-mediated autoimmune conditions. Using imlifidase is a novel approach to eliminate pathogenic IgG. It has a rapid onset of action, cleaving IgG-antibodies and inhibiting their reactivity within hours after administration. Hansa Biopharma has completed the Phase II stage of clinical development in patients with severe anti-GBM (Glomerular Basement Membrane) disease and decided to begin the Phase III clinical trial of an antibody-cleaving enzyme, imlifidase, for the treatment of anti-Glomerular Basement Membrane (anti-GBM) disease, the trial is expected to enroll about 50 patients, with recruitment of first patient in 2022. In 2018, Hansa Biopharma was granted orphan drug designation for imlifidase for Anti-GBM antibody disease in both the EU and the US.

Note: Detailed emerging therapies assessment will be provided in the final report.

Anti-Glomerular Basement Membrane (Anti-GBM) Disease Market Outlook

Anti-glomerular basement membrane (anti-GBM) disease is a life-threatening organ-specific autoimmune disorder. It causes the immune system to attack tissues in the lungs and kidneys and leads to rapid glomerulonephritis progression, with or without diffuse alveolar hemorrhage, and even respiratory failure. It happens when the immune system mistakenly attacks a protein called collagen because it recognizes it as a foreign substance. In anti-GBM disease, the body produces proteins (antibodies) that attach to the collagen in certain parts of the lungs and the kidneys. When they attach to the collagen, these antibodies cause severe inflammation and destruction of those tissues.

Classic cases of anti-GBM disease are diagnosed based on the presence of the anti-GBM antibody in serum samples and kidney or lung biopsy tissue samples. However, atypical cases of anti-GBM disease are also seen in clinical practice. Anti-GBM disease represents a rarely encountered pulmonary-renal disorder that manifests as the rapid progression of glomerulonephritis and alveolar hemorrhage. The pathogenesis of anti-GBM disease involves anti-GBM antibodies in the blood that target the α-3 chain of collagen IV in the glomerular and/or alveolar basement membranes. An elevated serum level of anti-GBM antibodies guides the diagnostic process. However, a tissue biopsy sample showing a linear pattern of antibody deposition along the basement membranes of kidney glomeruli, with or without alveoli showing this pattern of antibody deposition, is considered an important indicator confirming the diagnosis of anti-GBM disease. This is especially critical in patients with suspected anti-GBM disease whose serum is negative for anti-GBM antibodies.

The treatment of choice in anti-GBM disease is plasma exchange combined with prednisone and cyclophosphamide. Plasma exchange is able to remove anti-GBM antibodies along with other inflammation mediators while immunomodulators lower antibody formation. The initial prescription for plasma exchange usually includes a daily or alternating frequency of procedures with 4L of exchange during 2 or 3 weeks. In general, restoration is achieved with albumin except for those cases in which renal biopsy is required, where fresh plasma can be used to ensure that coagulation factors are potentially removed by plasma exchange. Plasma exchange must be accompanied by administration of glucocorticoids (usually methylprednisolone 15-30 mg/kg with a maximum dose of 1000 mg daily during 3 days followed by prednisone 1 mg/kg/day with a maximum dose of 60-80mg/day) and cyclophosphamide (with plans that include oral administration at doses of 2 mg/kg/day or intravenous administration). The length of the treatment is unknown. Usually, after the induction period with glucocorticoids and cyclophosphamide, treatment is maintained for at least 6 to 9 months with prednisone and azathioprine. Most of the reported patients in lengthy series were treated successfully with plasma exchange for 2 or 3 weeks followed by cyclophosphamide and glucocorticoids for the following 3 months and glucocorticoids alone for 6 to 9 months afterwards.

Anti-GBM antibody levels must be monitored weekly or twice weekly until negativity is reached and every 6 months thereafter in order to confirm sustained remission in the absence of clinical signs of recurrence. The presence of signs of recurrence along with positivity for anti-GBM antibodies is sufficient reason to justify a new cycle of plasma exchange. Sustained positivity of anti-GBM antibodies requires consideration of prolonging immunosuppressive therapy over time, usually with prednisone in decreasing doses and azathioprine in doses of 1-2 mg/kg/day. Renal recovery is unlikely in those dialysis-dependent patients. In this context (and in the absence of pulmonary hemorrhage), the risks of treatment with plasma exchange may exceed the benefits. Patients who are dialysis-dependent may benefit from a short cycle of combined therapy with plasma exchange and immunosuppressive therapy.

There is a high unmet need for new therapies for the treatment of anti-GBM disease, which is apparent by the unavailability of any approved drugs in the anti-GBM disease market. The current treatment consists of only one option of plasmapheresis with corticosteroid and prednisone which has been available in the market for some time. Also, due to the rarity of the disease there are some limitations that are required to improve for the development of the anti-GBM disease market. Some of the major unmet needs include the delay in diagnosis of the disease, although, there has been increasing awareness about atypical anti-GBM nephritis and complex clinical variants. Despite the many available options for diagnostic tests, the delayed establishment of the correct diagnosis and thus transfer to treatment centers results in many patients requiring permanent renal replacement therapy. Moreover, anti-GBM therapy is highly effective when detected at an early stage, making a high degree of awareness necessary to find these rare cases. Unawareness or diminished awareness is present when a patient’s perception of obvious disease manifestations differs from that of objective observers.

In short, efficacious pharmacologic options for managing the most incident and most disabling phases of anti-GBM are extremely limited. Treatments that work in this disorder are especially rare, therefore, new treatments are desperately needed.

Key Findings

The Anti-GBM Disease total market size in the 7MM is expected to rise by 2030 with a CAGR of 22.2% during the study period (2018-2030). According to the estimates, the highest market size of Anti-GBM found in the United States.

The United States Market Outlook

The total market size of Anti-GBM Disease in the United States is expected to increase with a CAGR of 20.0% in the study period (2018-2030).

EU-5 Countries: Market Outlook

The total market size of Anti-GBM Disease in EU-5 countries is expected to increase with a CAGR of 29.1% in the study period (2018-2030).

Japan Market Outlook

The total market size of Anti-GBM Disease in Japan is expected to decline with a CAGR of -0.4% in the study period (2018-2030).

Anti-Glomerular Basement Membrane (Anti-GBM) Disease Pipeline Development Activities

The drugs which are in pipeline include:

1. Idefirix (Imlifidase/HMed-IdeS): Hansa Biopharma

Note: Detailed emerging therapies assessment will be provided in the final report.

Analyst Commentary

• Increase in the geriatric population play an important role in the development and exacerbation of Anti-GBM therapeutics market. Due to the growing pace of the elder population, the market of Anti-GBM is expected to increase in the forecast period.
• Despite having lots of diagnosis options there are high chances of patients getting delay in diagnosis for Anti-GBM can be observed and the possible hurdles might be the lack of proper understanding of the disease (Anti-GBM).
• Among the emerging therapies, Hansa Biopharma’s Idefirix (Imlifidase/HMed-IdeS), appears to be the only drug which have the potential to transform the Anti-GBM Disease market owing to impressive clinical data.

Access and Reimbursement Scenario in Anti-GBM disease
Anti-Glomerular Basement Membrane disease (anti-GBM) is a disease that occurs as a result of injury to small blood vessels (capillaries) in the kidneys and/or lungs. Anti-GBM disease is an autoimmune disorder. It occurs when the immune system mistakenly attacks and destroys healthy body tissue. People with this syndrome develop substances that attack a protein called collagen in the tiny air sacs in the lungs and the filtering units (glomeruli) of the kidneys. Autoantibodies are antibodies directed toward the body itself (rather than towards something foreign such as bacteria or viruses). In anti-GBM disease, these autoantibodies are targeted to the basement membrane, which is part of the wall of these capillary blood vessels in the kidneys and lungs. The name anti-GBM disease reflects the fact that this disease is caused by autoantibodies targeting and causing damage to (anti-) the glomerular basement membrane (GBM).

Standard treatment for anti-GBM disease includes plasmapheresis, to rapidly remove pathogenic autoantibody, along with cyclophosphamide and corticosteroids, to inhibit further autoantibody production and to ameliorate end-organ inflammation. The use of this combination of therapies was first described in 1976, and they remain the core recommendation of the latest Kidney Disease Improving Global Outcomes guideline for treating anti-GBM glomerulonephritis. The inclusion of plasmapheresis is supported by observational studies that suggest improved renal and patient survival compared with historical cohorts treated with immunosuppression alone.

A combination of therapeutic plasma exchange (or immunoadsorption), cyclophosphamide, and glucocorticoids is considered standard of care management, but despite early initiation, patients with poor prognostic factors often remain dialysis dependent. Imlifidase (IdeS), capable to cleave IgG within hours, has been tested in a phase II trial.

In June 2019, the US FDA approved Soliris (eculizumab) injection for intravenous use for the treatment of NMOSD in adult patients who are anti-aquaporin-4 (AQP4) antibody positive. Soliris was the first FDA-approved treatment for NMOSD, a debilitating disease that profoundly impacts patients’ lives. The High Authority of Health (HAS) in France, recommends that treatment with Soliris (eculizumab) be administered in a multiple sclerosis resource and expertise center or in a center specializing in rare inflammatory brain and spinal cord diseases, with prescription restricted to neurologists in the context of a multidisciplinary review meeting justified in view of the risk of using SOLIRIS (eculizumab) outside the reimbursement scope defined by the Committee on the basis of available efficacy and safety data, particularly first-line use or use in patients who do not have anti-AQP4 antibodies (off-label).

Since, there are no approved therapies for the treatment of AGBM currently available in the market. The reimbursement scenario can only be estimated. But looking at the evidences from the reimbursement provided to patients for Soliris in the treatment of NMOSD, it can estimated that the reimbursement for any approved treatment in AGBM might be provided. Moreover, Imlifidase was granted Orphan Drug Designation in anti-GBM disease by both the FDA and the European Commission in 2018. Therefore, there is a high possibility that reimbursement will be granted for the treatment of AGBM after its approval.

Note: Detailed HTA assessment will be provided in the final report.

KOL-Views

To keep up with current market trends, we take KOLs and SME’s opinion working in the Anti-GBM domain through primary research to fill the data gaps and validate our secondary research. Their opinion helps to understand and validate current and emerging therapies treatment patterns or Anti-GBM market trends. This will support the clients in potential upcoming novel treatment by identifying the overall scenario of the market and the unmet needs.

Competitive Intelligence Analysis

We perform Competitive and Market Intelligence analysis of the Anti-GBM Market by using various Competitive Intelligence tools that includes - SWOT analysis, PESTLE analysis, Porter’s five forces, BCG Matrix, Market entry strategies, etc. The inclusion of the analysis entirely depends upon the data availability.

Scope of the Report

• The report covers the descriptive overview of Anti-GBM, explaining its causes, signs and symptoms, pathophysiology, and currently available therapies.
• Comprehensive insight has been provided into the Anti-GBM epidemiology and treatment in the 7MM.
• Additionally, an all-inclusive account of both the current and emerging therapies for Anti-GBM is provided, along with the assessment of new therapies, which will have an impact on the current treatment landscape.
• A detailed review of the Anti-GBM market; historical and forecasted is included in the report, covering drug outreach in the 7MM.
• The report provides an edge while developing business strategies, by understanding trends shaping and driving the global Anti-GBM market.

Report Highlights

• In the coming years, the Anti-GBM market is set to change due to the upcoming therapies with novel route of administrations which are under investigation and ongoing research in the Anti-GBM; which would expand the size of the market to enable the drug manufacturers to penetrate more into the market.
• The companies and academics are working to assess challenges and seek opportunities that could influence Anti-GBM R&D. The therapies under development are focused on novel approaches to treat/improve the disease condition.
• Major players are involved in developing therapies for Anti-GBM. The launch of emerging therapies will significantly impact the Anti-GBM market.
• A better understanding of disease pathogenesis will also contribute to the development of novel therapeutics for Anti-GBM.
• Our in-depth analysis of the pipeline assets across different stages of development (Phase III and Phase II), different emerging trends, and comparative analysis of pipeline products with detailed clinical profiles, key cross-competition, launch date along with product development activities will support the clients in the decision-making process regarding their therapeutic portfolio by identifying the overall scenario of the research and development activities.

Anti-Glomerular Basement Membrane (Anti-GBM) Disease Report Insights

• Patient Population
• Therapeutic Approaches
• Anti-GBM Pipeline Analysis
• Anti-GBM Market Size and Trends
• Market Opportunities
• Impact of upcoming Therapies

Anti-Glomerular Basement Membrane (Anti-GBM) Disease Report Key Strengths

• 10 Years Forecast
• 7MM Coverage
• Anti-GBM Epidemiology Segmentation
• Key Cross Competition
• Highly Analyzed Market

Drugs Uptake

Anti-Glomerular Basement Membrane (Anti-GBM) Disease Report Assessment

• SWOT Analysis
• Current Treatment Practices
• Unmet Needs
• Pipeline Product Profiles
• Conjoint Analysis
• Market Attractiveness
• Market Drivers and Barriers

Key Questions

Market Insights:

• What was the Anti-GBM Market share (%) distribution in 2018 and how it would look like in 2030?
• What would be the Anti-GBM total market size as well as market size by therapies across the 7MM during the study period (2018-2030)?
• What are the key findings of the market across the 7MM and which country will have the largest Anti-GBM market size during the study period (2018-2030)?
• At what CAGR, the Anti-GBM market is expected to grow in the 7MM during the study period (2018-2030)?
• What would be the Anti-GBM market outlook across the 7MM during the study period (2018-2030)?
• What would be the Anti-GBM market growth till 2030 and what will be the resultant market size in the year 2030?
• How would the market drivers, barriers, and future opportunities affect the market dynamics and subsequent analysis of the associated trends?
• Anti-GBM patient types/pool where unmet need is more and whether emerging therapies will be able to address the residual unmet need?
• How emerging therapies are performing on the parameters like efficacy, safety, route of administration (RoA), treatment duration, and frequencies based on their clinical trial results?
• Among the emerging therapies, what are the potential therapies which are expected to disrupt the Anti-GBM market?

Epidemiology Insights:

• What are the disease risks, burdens, and unmet needs of the Anti-GBM?
• What is the historical Anti-GBM patient pool in the seven major markets covering the United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan?
• What would be the forecasted patient pool of Anti-GBM in the 7 major markets covering the United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan?
• What will be the growth opportunities in the 7MM concerning the patient population about Anti-GBM?
• Out of all the 7MM countries, which country would have the highest incident population of Anti-GBM during the study period (2018-2030)?
• At what CAGR the population is expected to grow in the 7MM during the study period (2018-2030)?
• What are the various recent and upcoming events which are expected to improve the diagnosis of Anti-GBM?

Current Treatment Scenario and Emerging Therapies:

• What are the current options for the treatment of Anti-GBM?
• What are the current treatment guidelines for the treatment of Anti-GBM in the US, Europe, and Japan?
• How many companies are developing therapies for the treatment of Anti-GBM?
• How many therapies are developed by each company for the treatment of Anti-GBM?
• How many emerging therapies are in the mid-stage and late stages of development for the treatment of Anti-GBM?
• What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Anti-GBM therapies?
• What are the recent novel therapies, targets, mechanisms of action, and technologies developed to overcome the limitation of existing therapies?
• What are the clinical studies going on for Anti-GBM and their status?
• What are the key designations that have been granted for the emerging therapies for Anti-GBM?
• What is the global historical and forecasted market of Anti-GBM?

Reasons to buy

• The report will help in developing business strategies by understanding trends shaping and driving the Anti-GBM market.
• To understand the future market competition in the Anti-GBM market and Insightful review of the key market drivers and barriers.
• Organize sales and marketing efforts by identifying the best opportunities for Anti-GBM in the US, Europe (Germany, France, Italy, Spain, and the United Kingdom), and Japan.
• Identification of strong upcoming players in the market will help in devising strategies that will help in getting ahead of competitors.
• Organize sales and marketing efforts by identifying the best opportunities for the Anti-GBM market.
• To understand the future market competition in the Anti-GBM market.