Epidermolysis Bullosa Market Insight, Epidemiology And Market Forecast - 2034

Epidermolysis Bullosa Market Insight, Epidemiology And Market Forecast - 2034

Key Highlights

Alport syndrome is an inherited disease, with X-linked being the most common type of it, and accounts for approximately 80% of the total cases.

In the absence of intervention, approximately 90% of males experience kidney failure by the age of 40, whereas females less commonly and more gradually progress to kidney failure.

The majority of Alport syndrome patients remain undiagnosed due to the asymptomatic nature of the disease and misdiagnosis.

ACE inhibitors and ARBs form the current mainstay treatment for CKD in Alport syndrome. However, in the past few years, interest in developing new treatments has increased significantly. There are now several well-established patient-founded organizations specific to Alport syndrome to help facilitate, develop, and aid in the progression of clinical trials, hoping to lead to the discovery of new and novel treatments for Alport syndrome.

Although treatment may slow the progression of kidney disease in Alport syndrome, there is no cure for the disorder and no treatment has thus far been shown to completely stop kidney decline

Over the next decade, improved early diagnosis and treatment advancements are expected to raise the age at which kidney failure manifests in individuals with Alport syndrome. Additional benefits are anticipated from novel therapies that can complement ACE inhibition. While safe and effective curative therapies are within the realm of possibility, notable challenges must be addressed to transform these possibilities into reality.

The late-stage pipeline of Alport syndrome is not that strong with several therapies only, including a small molecule gene therapy ELX-02 (Eloxx Pharmaceuticals) for patients with a nonsense mutation only; other therapies are Atrasentan (Novartis) and Finerenone (Bayer). Setanaxib by Calliditas Therapeutics completed its preclinical trial and is now planning for Phase II, while Bayer has already started a Phase I trial of Semaphorin-3A in healthy subjects.

ELX-02 can potentially become the first gene therapy for alport syndrome patients with nonsense mutations.

The total number of prevalent cases of alport syndrome in the US were ~67,900 in 2023.

In 2023, the US accounted for the maximum share of the total market in the 7MM, i.e.,approximately USD 11 million, followed by Germany.

DelveInsight's “Alport Syndrome – Market Insights, Epidemiology, and Market Forecast – 2034” report delivers an in-depth understanding of Alport Syndrome , historical and forecasted epidemiology as well as the Alport Syndrome market trends in the US, EU4 (Germany, Spain, Italy, and France) and the United Kingdom, and Japan.

Alport Syndrome market report provides real-world prescription pattern analysis, emerging drugs, market share of individual therapies, and historical and forecasted 7MM Alport Syndrome market size from 2020 to 2034. The report also covers current Alport Syndrome treatment practices/algorithms and unmet medical needs to curate the best opportunities and assess the market’s potential.

Geography Covered

The United States

EU4 (Germany, France, Italy, and Spain) and the United Kingdom

Japan

Study Period: 2020–2034

Alport Syndrome Understanding and Treatment Algorithm

Alport Syndrome Overview and Diagnosis

Alport syndrome is a rare genetic disorder characterized by abnormalities of the inner ear and the eye. The disease is caused by an inherited defect in type IV collagen, a structural material needed for the normal function of different body parts. Alport syndrome can present itself in many forms. This includes X-linked Alport syndrome (XLAS), autosomal recessive Alport syndrome (ARAS), and autosomal dominant Alport syndrome (ADAS). Clinically, it is associated with microscopic hematuria, followed by proteinuria and chronic renal insufficiency with end-stage renal disease in young adults.

Diagnosis of Alport syndrome is much more likely when there is a family history of Alport syndrome, early hearing loss, hematuria, or kidney failure with unknown cause. Suspicion of the disease is generally heightened when hearing loss is present. Diagnosis can be confirmed by examination of kidney biopsies, where collagen expression and structural changes to the GBM can be observed by immunofluorescence and electron microscopy, respectively. In the case of X-linked Alport syndrome, immunofluorescence microscopy may be employed on skin biopsies to assess the presence of the type IV collagen a-5 chain. Diagnosis is increasingly confirmed by genetic testing.

Further details related to country-based variations in diagnosis are provided in the report…

Alport Syndrome Treatment

Unfortunately, there is no specific treatment for Alport syndrome. Treatment is focused on limiting the progression of proteinuria and kidney disease, often including renin–angiotensin–aldosterone system blockers. While these blockers lower blood pressure in Alport syndrome patients, their benefits are thought to derive from the antifibrotic effects of these agents. Other nonspecific treatments used in Alport syndrome are diuretics and dietary interventions. Although the treatment may delay the onset of renal impairment, most people affected by Alport will ultimately require dialysis or a kidney transplant.

Medications known as angiotensin-converting enzyme (ACE) inhibitors have been used to treat individuals with Alport syndrome. Historical data strongly suggests that early treatment with ACE inhibitors can delay progression to end-stage renal disease in males and females with Alport syndrome. Some individuals do not respond to or cannot tolerate ACE inhibitors. These individuals may be treated with drugs known as angiotensin receptor blockers (ARBs).

Alport Syndrome Epidemiology

The Alport Syndrome epidemiology chapter in the report provides historical as well as forecasted epidemiology segmented in the 7MM covering the United States, EU4 countries (Germany, France, Italy, and Spain) and the United Kingdom, and Japan from 2020 to 2034.

The total number of prevalent cases of alport syndrome in the 7MM were ~159,000 in 2023.

Alport syndrome is more prevalent in adult population with ~86% contribution in the 7MM, while pediatric population accounted for ~14% cases.

The X-linked alport syndrome (XLAS) is most prevalent subtype of Alport syndrome with around 11,500 cases in 2023 in the US while autosomal dominant alport syndrome (ADAS) being the least prevalent subtype of alport syndrome.

In EU4 region, the total prevalent cases were the highest in Germany, with nearly 17,000 cases in 2023, while Spain had the least number of cases in 2023.

Alport syndrome affects both men and women in the United States with minimal disparity, although men are slightly more impacted than women.

Alport Syndrome Drug Chapters

The drug chapter segment of the alport syndrome report encloses a detailed analysis of alport syndrome marketed drugs and late-stage (Phase III and Phase II) pipeline drugs. It also deep dives into the alport syndrome pivotal clinical trial details, recent and expected market approvals, patent details, the latest news, and recent deals and collaborations.

Emerging Drugs

ELX-02: Eloxx Pharmaceuticals

Eloxx’s lead investigational product candidate, ELX-02, is a small molecule drug candidate designed to restore the production of full-length functional proteins. It is a synthetic aminoglycoside administered through the subcutaneous route and inhalational route. It optimizes ribosomal read-through of premature termination codons (PTC), through which full-length functional proteins can be produced. It acts by targeting CFTR. Preclinical studies support ELX-02 activity in nonsense mutation genetic kidney diseases.

Based on the positive results from the Phase II trial, Eloxx intends to gain alignment with the FDA on the design of a pivotal trial for ELX-02 for the treatment of Alport syndrome with nonsense mutations and the potential for seeking Breakthrough Therapy Designation.Currently, ELX-02 is in Phase II clinical development for the treatment of Alport syndrome in patients with nonsense mutations.

Atrasentan: Chinook Therapeutics/Novartis

Atrasentan is a potent and selective endothelin A (ETA) receptor antagonist that has the potential to provide benefit in multiple chronic kidney diseases such as IgA nephropathy (IgAN) and other proteinuric glomerular diseases by reducing proteinuria and having direct anti-inflammatory and anti-fibrotic effects to preserve kidney function. The company in-licensed atrasentan from AbbVie in December 2019, which previously developed atrasentan for diabetic kidney disease (DKD).

Currently, the company is evaluating the drug in Phase II AFFINITY study in additional proteinuric glomerular diseases. The basket trial has four cohorts consisting of patients with IgAN with urine protein to creatinine ratio (UPCR) of 0.5 to less than 1.0 g/g, focal segmental glomerulosclerosis (FSGS), Alport syndrome, and diabetic kidney disease (DKD).

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

Drug Class Insights

There is a growing focus on sodium–glucose cotransporter-2 (SGLT2) inhibitors as potential agents to decelerate the progression of chronic kidney disease. Due to their existing approval for hypoglycemic effects, there is considerable interest in the off-label use of these agents for individuals with Alport syndrome. ACE inhibitors and ARBs form the current mainstay treatment for CKD in Alport syndrome. However, in the past few years, interest in developing new treatments has increased significantly. The late-stage pipeline of Alport syndrome is not that strong with few therapies only, including a small molecule gene therapy ELX-02 (Eloxx Pharmaceuticals) for patients with a nonsense mutation only; other therapies are Atrasentan (Novartis) and Finerenone (Bayer). Setanaxib by Calliditas Therapeutics completed its preclinical trial and is now planning for Phase II, while Bayer has already started a Phase I trial of Semaphorin-3A in healthy subjects.

Alport Syndrome Market Outlook

Alport syndrome comes with many challenges and lacks real-world treatment prescription studies, but some researchers have tried their best to make the utilization of drugs a bit clear. As there is no approved therapy for this condition, the treatment is often off-label. National Kidney Foundation and the Alport Syndrome Foundation published a document and discussed this condition. It was found that approximately 52% of the patients receive ACE/ARB, and various other medications such as a statin, Allopurinol, antidepressants, and others are also prescribed to manage the symptoms, and many patients also use drug–device combinations.

There is an urgent need for promising therapies to address the significant burden. Companies including Eloxx Pharmaceuticals, Chinook Therapeutics (A Novartis Company), Bayer, Calliditas Therapeutics, Evotec and others are investigating their potential drug candidates that can significantly change the market landscape during the forecast period, including ELX-02, Atrasentan, Finerenone, setanaxib, BAY3401016, and others. The upcoming drugs, such as Eloxx’s ELX-02 is expected to be the first potential approval (Gene therapy) for treating patients with Alport Syndrome, followed by Novartis’ Atrasentan, which is considered as another promising therapy with a strong efficacy. Introducing these promising therapies holds the potential to bring a significant ray of hope to patients grappling with this condition. The advent of such molecules is expected to alter the treatment paradigm substantially.

The total market size in the 7MM for Alport syndrome was estimated to be ~USD 20 million in 2023, which is expected to show positive growth by 2034.

Among EU4 and the UK, Germany accounted for the largest market size in 2023.

There is a growing focus on sodium–glucose cotransporter-2 (SGLT2) inhibitors as potential agents to decelerate the progression of chronic kidney disease. Due to their existing approval for hypoglycemic effects, there is considerable interest in the off-label use of these agents for individuals with Alport syndrome.

Alport syndrome registries in Europe and France established the efficacy of RAAS inhibition for delaying kidney failure in people with Alport syndrome.

Alport Syndrome Drugs Uptake

This section focuses on the uptake rate of potential drugs expected to be launched in the market during 2024–2034, which depends on the competitive landscape, safety, efficacy data, and order of entry. It is important to understand that the key players evaluating their novel therapies in the pivotal and confirmatory trials should remain vigilant when selecting appropriate comparators to stand the greatest chance of a positive opinion from regulatory bodies, leading to approval, smooth launch, and rapid uptake.

There is an urgent need for promising therapies to address the significant burden. Companies including Reata Pharmaceuticals, Regulus Therapeutics, and many more are investigating their potential candidate and potential drugs that can significantly change the market landscape during the forecast period, including ELX-02, Atrasentan, Finerenone, setanaxib, BAY3401016, and others. The upcoming drug Atrasentanis is considered one of the promising drugs, and it belongs to the family of drugs called endothelin-1 protein receptor antagonists. It is a novel, selective endothelin A receptor antagonist.

Introducing these promising therapies holds the potential to bring a significant ray of hope to patients grappling with this condition. The advent of such molecules is expected to alter the treatment paradigm substantially.

Further detailed analysis of emerging therapies drug uptake in the report…

Alport Syndrome Activities

The report provides insights into therapeutic candidates in Phase III and 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 Alport Syndrome emerging therapies.

KOL Views

To keep up with the real-world scenario in current and emerging market trends, we take opinions from Key Industry leaders working in the domain through primary research to fill the data gaps and validate our secondary research. Industry Experts contacted for insights on the evolving treatment landscape, patient reliance on conventional therapies, patient’s therapy switching acceptability, and drug uptake along with challenges related to accessibility, including Medical/scientific writers, Professors and Others.

DelveInsight’s analysts connected with 20+ KOLs to gather insights; however, interviews were conducted with 12+ KOLs in the 7MM. Centers such as University of Manchester, United Kingdom, University of Louisville School of Medicine and others were contacted. Their opinion helps understand and validate current and emerging therapy treatment patterns or Alport Syndrome market trends.

Qualitative Analysis

We perform qualitative and market Intelligence analysis using various approaches, such as SWOT analysis. In the SWOT analysis, strengths, weaknesses, opportunities, and threats in terms of gaps in disease diagnosis, patient awareness, physician acceptability, competitive landscape, cost-effectiveness, and geographical accessibility of therapies are provided.

Market Access and Reimbursement

The reimbursement landscape for therapies is characterized by intricacies and variations across different countries. With high initial costs associated with the development and manufacturing of therapies, reimbursement models often adopt a value-based approach, considering the long-term benefits and potential cost savings compared to traditional treatments. Health Technology Assessments (HTAs) are commonly employed to evaluate clinical effectiveness, cost-effectiveness, and overall value, taking into account factors like disease severity and the availability of alternative treatments.

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.

Scope of the Report

The report covers a segment of key events, an executive summary, and a descriptive overview of alport syndrome, explaining its causes, signs and symptoms, pathogenesis, and currently available therapies.

Comprehensive insight has been provided into the epidemiology segments and forecasts, the future growth potential of diagnosis rate, disease progression, and treatment guidelines.

Additionally, an all-inclusive account of the current and emerging therapies, along with the elaborative profiles of late-stage and prominent therapies, will impact the current treatment landscape.

A detailed review of the alport syndrome market, historical and forecasted market size, market share by therapies, detailed assumptions, and rationale behind our approach is included in the report, covering the 7MM drug outreach.

The report provides an edge while developing business strategies by understanding trends through SWOT analysis and expert insights/KOL views, patient journey, and treatment preferences that help shape and drive the 7MM.

Alport Syndrome Report Insights

Patient Population

Therapeutic Approaches

Alport Syndrome Pipeline Analysis

Alport Syndrome Market Size and Trends

Existing and future Market Opportunity

Alport Syndrome Report Key Strengths

11 Years Forecast

7MM Coverage

Alport Syndrome Epidemiology Segmentation

Key Cross Competition

Conjoint analysis

Drugs Uptake and Key Market Forecast Assumptions

Alport Syndrome Report Assessment

Current Treatment Practices

Unmet Needs

Pipeline Product Profiles

Market Attractiveness

Qualitative Analysis (SWOT and Conjoint Analysis)

FAQs

What is the historical and forecasted alport syndrome patient pool in the US, EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan?

What was the alport syndrome total market size, the market size by therapies, market share (%) distribution in 2023, and what would it look like in 2034? What are the contributing factors for this growth?

What will be the impact of gene therapy on other emerging therapies?

What are the pricing variations among different geographies for approved and off-label therapies?

How would the market drivers, barriers, and future opportunities affect the market dynamics and subsequent analysis of the associated trends?

Although multiple expert guidelines recommend testing for targetable mutations prior to therapy initiation, why do barriers to testing remain high?

What are the current and emerging options for treating alport syndrome?

How many companies are developing therapies to treat alport syndrome ?

What are the recent novel therapies, targets, mechanisms of action, and technologies developed to overcome the limitations of existing therapies?

Patient acceptability in terms of preferred treatment options as per real-world scenarios?

Reasons to buy

The report will help develop business strategies by understanding the latest trends and changing treatment dynamics driving the alport syndrome market.

Insights on patient burden/disease prevalence, evolution in diagnosis, and factors contributing to the change in the epidemiology of the disease during the forecast years.

Understand the existing market opportunities in varying geographies and the growth potential over the coming years.

Distribution of historical and current patient share based on real-world prescription data in the US, EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan.

Identifying strong upcoming players in the market will help devise strategies to help get ahead of competitors.

Highlights of access and reimbursement policies, barriers to accessibility of expensive off-label therapies, and patient assistance programs.

To understand Key Opinion Leaders’ perspectives around the accessibility, acceptability, and compliance-related challenges of existing treatment to overcome barriers in the future.

Detailed insights on the unmet needs of the existing market so that the upcoming players can strengthen their development and launch strategy.