The dynamics of the Alport syndrome market are anticipated to change in the coming years owing to the improvement in the rise in healthcare spending across the world.
LAS VEGAS, April 17, 2024 /PRNewswire/ -- DelveInsight's Alport Syndrome Market Insights report includes a comprehensive understanding of current treatment practices, Alport syndrome emerging drugs, market share of individual therapies, and current and forecasted market size from 2020 to 2034, segmented into 7MM [the United States, the EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan].
Key Takeaways from the Alport Syndrome Market Report
- According to DelveInsight's analysis, the market size for Alport syndrome reached USD 20 million in 2023 across the 7MM.
- Alport syndrome is an inherited disease, with X-linked being the most common type of it, and accounts for approximately 85% of the total cases.
- DelveInsight's analysis reveals that the overall prevalent population of Alport syndrome in the 7MM was reported as 159K in 2023.
- Leading Alport syndrome companies such as Eloxx Pharmaceuticals, Chinook Therapeutics (A Novartis company), Bayer, Calliditas Therapeutics, Evotec, and others are developing novel Alport syndrome drugs that can be available in the Alport syndrome market in the coming years.
- Some promising Alport syndrome therapies in the pipeline include ELX-02, Atrasentan, Finerenone, Setanaxib, BAY3401016, and others.
- 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.
- ELX-02 can potentially become the first gene therapy for Alport syndrome patients with nonsense mutations.
Discover which therapies are expected to grab the major Alport syndrome market share @ Alport Syndrome Market Report
Alport Syndrome Overview
Alport syndrome denotes a range of hereditary, diverse conditions impacting the kidney's basement membranes, often with effects on the cochlea and eyes. It is a genetic condition resulting from mutations in the genes that code for alpha-3, alpha-4, and alpha-5 of type 4 collagen (COL4A3, COL4A4, COL4A5), also known as the collagen 4 a345 network. The disorder manifests in different forms such as autosomal recessive Alport syndrome (ARAS), X-linked Alport syndrome (XLAS), and autosomal dominant Alport syndrome (ADAS).
People with Alport syndrome may exhibit signs such as blood in urine, excessive protein in urine, swelling, high blood pressure, progressive deterioration in kidney function, and ultimately, end-stage renal disease (ESRD). As time progresses, these symptoms tend to worsen, with increasing protein in the urine, elevated blood pressure, declining glomerular filtration rate (GFR), and eventual development of ESRD. The diagnostic methods for confirming Alport syndrome have been advancing in recent years. Diagnosis typically involves recognizing characteristic symptoms, a thorough review of the patient's medical history, and a comprehensive clinical assessment. Suspicions of Alport syndrome are heightened in individuals with a family history of the condition, unexplained kidney failure, early onset of hearing loss, or the presence of blood in urine.
Alport Syndrome Epidemiology Segmentation
Alport syndrome is more prevalent in the adult population with ~86% contribution in the 7MM, while the pediatric population accounted for ~14% of cases.
The X-linked Alport syndrome (XLAS) is the most prevalent subtype of Alport syndrome with around 11K cases in 2023 in the US while autosomal dominant Alport syndrome (ADAS) is the least prevalent subtype of Alport syndrome.
The Alport syndrome market report proffers epidemiological analysis for the study period 2020-2034 in the 7MM segmented into:
- Total Prevalent Cases of Alport Syndrome
- Total Diagnosed Prevalent Cases of Alport Syndrome
- Gender-specific Cases of Alport Syndrome
- Age-specific Cases of Alport Syndrome
- Type-specific Cases of Alport Syndrome
Alport Syndrome Treatment Market
Treatment for Alport syndrome typically focuses on managing symptoms and slowing the progression of kidney damage. This often involves a multidisciplinary approach with nephrologists, otolaryngologists, and ophthalmologists. Medications such as ACE inhibitors and angiotensin II receptor blockers (ARBs) are commonly prescribed to help control high blood pressure and reduce proteinuria, thus preserving kidney function. In cases of advanced kidney failure, dialysis or kidney transplantation may be necessary to replace lost kidney function and improve quality of life. Ongoing research into potential gene therapies and other innovative treatments holds promise for the future of Alport syndrome management.
Additionally, supportive care plays a crucial role in managing Alport syndrome. This includes regular monitoring of kidney function, hearing, and vision, as well as lifestyle modifications such as maintaining a healthy diet, staying hydrated, and avoiding nephrotoxic substances. Genetic counseling is often recommended for families to understand the inheritance pattern of Alport syndrome and make informed decisions. By addressing both the medical and emotional aspects of the condition, individuals with Alport syndrome can work with their healthcare team to optimize their treatment plan and lead fulfilling lives despite the challenges posed by this rare genetic disorder.
To know more about Alport syndrome treatment guidelines, visit @ Alport Syndrome Management
Alport Syndrome Pipeline Therapies and Key Companies
- ELX-02: Eloxx Pharmaceuticals
- Atrasentan: Chinook Therapeutics/Novartis
- Finerenone: Bayer
- Setanaxib: Calliditas Therapeutics
- BAY3401016: Evotec/Bayer
Learn more about the FDA-approved drugs for Alport syndrome @ Drugs for Alport Syndrome Treatment
Alport Syndrome Market Dynamics
The dynamics of the Alport syndrome market are expected to change in the coming years. With a deeper understanding of the genetic basis of Alport syndrome, personalized treatment strategies can be tailored to an individual's specific genetic profile. ELX-02 is expected to garner the first mover advantage in Alport syndrome apart from being the first gene therapy with reduced frequency of administration (8 weeks), as an increase in research and development has paved the way for discovering novel MoA therapies such as dual NOX inhibitor (setanaxib), semaphorin-3A blocker (BAY3401016), selective endothelin A receptor antagonist (atrasentan), amidst the usage of ACE/ARB inhibitors as standard of care.
Genetic advances may open the way for personalized treatment tailored to the particular genetic profiles of individuals with Alport syndrome, thus there is a huge opportunity for precision medicine approaches, with patient advocacy groups such as the Alport Syndrome Foundation playing a pivotal role in shaping the landscape for Alport syndrome by directing the majority of its resources to research and research-related activities, resulting in groundbreaking knowledge and clinical trials pertaining to Alport syndrome.
Furthermore, many potential therapies are being investigated for the treatment of Alport syndrome, and it is safe to predict that the treatment space will significantly impact the Alport syndrome market during the forecast period. Moreover, the anticipated introduction of emerging therapies with improved efficacy and a further improvement in the diagnosis rate are expected to drive the growth of the Alport syndrome market in the 7MM.
However several factors may impede the growth of the Alport syndrome market. The gene mutation causing Alport syndrome might remain silent in a person's parents, complicating diagnosis, often resulting in late-stage identification following significant kidney damage. Over half of those under current therapies experience only moderate symptom reduction, leaving substantial needs unmet. Eloxx Pharmaceuticals' gene therapy is set to launch at premium prices, potentially raising accessibility and affordability concerns in certain nations. Off-label treatments such as ACE inhibitors and ARBs pose a significant threat to the Alport syndrome market's overall growth.
Emerging therapies, with their high treatment costs, could further limit market access, rendering drugs, specialized care, and potential renal replacement unaffordable for many families. Financial constraints might hinder patients' critical therapy access, a problem compounded by various FDA trial discontinuations and approval withdrawals. In 2022, Bardoxolone Methyl faced FDA rejection, and its Japanese trial was halted, while Sanofi discontinued Lademirsen's Phase II trial. In addition, the undiagnosed, unreported cases and the unawareness about the disease may also impact the Alport syndrome market growth.
Alport Syndrome Market Report Metrics | Details |
Study Period | 2020-2034 |
Coverage | 7MM [the United States, the EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan]. |
Alport Syndrome Market CAGR | 69 % |
Alport Syndrome Market Size in 2023 | USD 20 Million |
Key Alport Syndrome Companies | Eloxx Pharmaceuticals, Chinook Therapeutics/Novartis, Bayer, Calliditas Therapeutics, Evotec, and others |
Key Pipeline Alport Syndrome Therapies | ELX-02, Atrasentan, Finerenone, Setanaxib, BAY3401016, and others |
Scope of the Alport Syndrome Market Report
- Therapeutic Assessment: Alport Syndrome current marketed and emerging therapies
- Alport Syndrome Market Dynamics: Key Market Forecast Assumptions of Emerging Alport Syndrome Drugs and Market Outlook
- Competitive Intelligence Analysis: SWOT analysis and Market entry strategies
- Unmet Needs, KOL's views, Analyst's views, Alport Syndrome Market Access and Reimbursement
Discover more about Alport syndrome drugs in development @ Alport Syndrome Clinical Trials
Table of Contents
1 | Key Insights |
2 | Report Introduction |
3 | Executive Summary of Alport Syndrome |
4 | Epidemiology and Market Forecast Methodology |
5 | Key Events |
6 | Alport Syndrome Market Overview at a Glance |
6.1 | Market Size Distribution by Therapies in 2023 |
6.2 | Market Size Distribution by Therapies in 2034 |
7 | Disease Background and Overview |
7.1 | Introduction |
7.2 | Types of Alport Syndrome |
7.3 | Signs and Symptoms |
7.3.1 | Renal Manifestations |
7.3.2 | Hearing Impairment |
7.3.3 | Ocular Manifestations |
7.3.4 | Leiomyomatosis |
7.4 | Causes of Alport Syndrome |
7.5 | Pathophysiology of Alport Syndrome |
7.6 | Diagnosis of Alport Syndrome |
7.6.1 | Clinical Diagnosis |
7.6.2 | Genetic Diagnosis |
7.6.3 | Other Tests |
7.7 | Prognosis |
7.8 | Differential Diagnosis |
7.9 | Prognosis |
8 | Treatment of Alport Syndrome |
8.1 | Treatment Algorithm |
9 | Diagnostic and Treatment Guidelines |
9.1 | Guidelines for Genetic Testing and Management of Alport Syndrome |
9.2 | Clinical Practice Recommendations for the Diagnosis and Management of Alport Syndrome in Children, Adolescents, and Young Adults (2020) |
9.3 | Expert Guidelines for the Management of Alport Syndrome and Thin Basement Membrane Nephropathy: American Society of Nephrology (2013) |
10 | Epidemiology and Patient Population |
10.1 | Key Findings |
10.2 | Assumptions and Rationale |
10.3 | Total Prevalent Cases of Alport Syndrome in the 7MM |
10.4 | Total Diagnosed Prevalent Cases of Alport Syndrome in the 7MM |
10.5 | The United States |
10.5.1 | Total Prevalent Cases of Alport Syndrome in the United States |
10.5.2 | Total Diagnosed Prevalent Cases of Alport Syndrome in the United States |
10.5.3 | Gender-specific Cases of Alport Syndrome in the United States |
10.5.4 | Age-specific Cases of Alport syndrome in the United States |
10.5.5 | Type-specific Cases of Alport Syndrome in the United States |
10.6 | EU4 and the UK |
10.6.1 | Total Prevalent Cases of Alport Syndrome in EU4 and the UK |
10.6.2 | Total Diagnosed Prevalent Cases of Alport Syndrome in EU4 and the UK |
10.6.3 | Gender-specific Cases of Alport Syndrome in EU4 and the UK |
10.6.4 | Age-specific Cases of Alport Syndrome in EU4 and the UK |
10.6.5 | Type-specific Cases of Alport syndrome in EU4 and the UK |
10.7 | Japan |
10.7.1 | Total Prevalent Cases of Alport Syndrome in Japan |
10.7.2 | Total Diagnosed Prevalent Cases of Alport Syndrome in Japan |
10.7.3 | Gender-specific Cases of Alport Syndrome in Japan |
10.7.4 | Age-specific Cases of Alport Syndrome in Japan |
10.7.5 | Type-specific Cases of Alport Syndrome in Japan |
11 | Patient Journey |
12 | Emerging Therapies |
12.1 | Key Cross Competition |
12.2 | ELX-02: Eloxx Pharmaceuticals |
12.2.1 | Product Description |
12.2.2 | Other Developmental Activity |
12.2.3 | Clinical Development |
12.2.4 | Safety and Efficacy |
12.3 | Atrasentan: Chinook Therapeutics/Novartis |
12.3.1 | Product Description |
12.3.2 | Other Developmental Activity |
12.3.3 | Clinical Development |
12.3.4 | Safety and Efficacy |
12.4 | Finerenone: Bayer |
12.4.1 | Product Description |
12.4.2 | Clinical Development |
12.4.3 | Safety and Efficacy |
12.5 | Setanaxib: Calliditas Therapeutics |
12.5.1 | Product Description |
12.5.2 | Other Developmental Activity |
12.6 | BAY3401016: Evotec/Bayer |
12.6.1 | Product Description |
12.6.2 | Other Developmental Activity |
13 | Alport Syndrome: 7MM Market Analysis |
13.1 | Key Findings |
13.2 | Market Outlook |
13.3 | Conjoint Analysis |
13.4 | Key Market Forecast Assumptions |
13.5 | Total Market Size of Alport Syndrome in the 7MM |
13.6 | United States Market Size |
13.6.1 | Total Market Size of Alport Syndrome in the United States |
13.6.2 | Market Size of Alport Syndrome by Therapies in the United States |
13.7 | EU4 and the UK Market Size |
13.7.1 | Total Market Size of Alport Syndrome in EU4 and the UK |
13.7.2 | Market Size of Alport Syndrome by Therapies in EU4 and the UK |
13.8 | Japan Market Size |
13.8.1 | Total Market Size of Alport Syndrome in Japan |
13.8.2 | Market Size of Alport Syndrome by Therapies in Japan |
14 | Unmet Needs |
15 | SWOT Analysis |
16 | KOL Views |
17 | Market Access and Reimbursement |
17.1 | United States |
17.1.1 | Centers for Medicare and Medicaid Services (CMS) |
17.2 | EU4 and the UK |
17.2.1 | Germany |
17.2.2 | France |
17.2.3 | Italy |
17.2.4 | Spain |
17.2.5 | United Kingdom |
17.3 | Japan |
17.3.1 | MHLW |
18 | Appendix |
18.1 | Bibliography |
18.2 | Report Methodology |
19 | DelveInsight Capabilities |
20 | Disclaimer |
21 | About DelveInsight |
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