Alport Syndrome Market Summary
The Alport syndrome market in the 7MM was valued at around USD 20 million in 2023 and is projected to grow by 2034. The disorder is primarily X-linked, accounting for 80% of cases, with 90% of males progressing to kidney failure by age 40 if untreated. In 2023, the US had about 67,900 prevalent cases, contributing the largest market share (~USD 11 million), followed by Germany. Growing efforts from patient-led organizations and increased R&D are driving clinical trials for novel therapies. The pipeline remains limited, with key candidates including ELX-02 (Eloxx Pharmaceuticals) for nonsense mutations, Atrasentan (Novartis), Finerenone (Bayer), Setanaxib (Calliditas Therapeutics), and Semaphorin-3A (Bayer). ELX-02 holds promise as the first gene therapy for certain patients.
DelveInsight’s report, “Alport Syndrome Market Insights, Epidemiology, and Market Forecast–2034”, offers a comprehensive analysis of Alport Syndrome, including historical and projected epidemiology, along with market trends across the United States, the EU5 (Germany, Spain, Italy, France, and the UK), and Japan. The study highlights current treatment practices, upcoming therapies, individual drug market shares, and the market size of Alport Syndrome from 2020 to 2034 across the seven major markets. It also examines treatment algorithms, key market drivers, barriers, and unmet needs, providing insights into growth opportunities and the overall potential of the Alport Syndrome market.
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Some facts of the Alport Syndrome Market Report are:
- DelveInsight projects that the Alport Syndrome market will expand at a CAGR of nearly 69% through 2034.
- In the 7MM, the Alport Syndrome market was valued at around USD 20 million in 2023, with expectations of steady growth by 2034.
- Major players in the Alport Syndrome market include Eloxx Pharmaceuticals, River 3 Renal Corp, Chinook Therapeutics, Travere Therapeutics, Reata Pharmaceuticals, among others.
- Upcoming therapies likely to enter the market are ELX-02, Atrasentan, Finerenone, Setanaxib, BAY3401016, and more.
- In April 2025, Travere Therapeutics reported results from a Phase 2 open-label study assessing the safety, efficacy, and pharmacokinetics of Sparsentan in pediatric patients with proteinuric glomerular diseases.
- In April 2025, Enyo Pharma released findings from a Vonafexor fixed dose-escalation study, focusing on safety and proof-of-concept in Alport Syndrome patients at risk of progression.
- In May 2025, Calliditas Therapeutics AB announced results from a Phase 2a randomized, double-blind, placebo-controlled trial of Setanaxib (a NOX1/4 inhibitor) evaluating its safety, tolerability, and preliminary efficacy in Alport Syndrome.
- In May 2024, Chinook Therapeutics shared outcomes from a Phase 2 basket study testing Atrasentan in patients with proteinuric glomerular diseases.
- In January 2024, ENYO Pharma received FDA approval for its IND application to initiate a Phase 2 trial of Vonafexor, a selective FXR agonist, for treating Alport Syndrome.
- In 2022, the 7MM recorded approximately 2,200 cases in children (2–11 years), 1,900 cases in adolescents (12–17 years), and 25,000+ cases in adults (18+ years).
- The Alport Syndrome market is projected to grow rapidly, driven by rising prevalence, increased awareness, and the introduction of advanced pipeline therapies.
- Across the 7MM, the total number of existing cases was estimated at ~160,000 in 2023, with forecasts showing an upward trend by 2034.
- The United States accounted for nearly 68,000 prevalent cases of Alport Syndrome in 2023.
- In the US (2023), cases were slightly male-dominant, with 52% males and 48% females.
- By type, around 11,500 cases were linked to X-linked Alport Syndrome (XLAS) in 2023.
Alport Syndrome Overview
Alport Syndrome is a genetic disorder affecting the kidneys, ears, and eyes, primarily caused by mutations in genes responsible for producing collagen, a crucial protein in the body’s connective tissues. This syndrome primarily manifests as progressive kidney disease, leading to renal failure in severe cases. Symptoms often include hematuria (blood in urine), proteinuria (protein in urine), high blood pressure, and hearing loss, typically starting in childhood or adolescence. Ocular abnormalities like lens dislocation and retinopathy may also occur. Diagnosis involves genetic testing, kidney biopsy, and evaluation of symptoms. Treatment aims to manage symptoms and slow kidney damage progression through medications controlling blood pressure and proteinuria. In advanced stages, kidney transplant or dialysis may be necessary. Regular monitoring and early intervention are vital in managing the condition and preventing complications.
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Alport Syndrome Market Outlook
The Alport Syndrome market size in the 7MM was ~USD 20 million in 2023, projected to grow by 2034, with Germany leading the EU4+UK market share. Meanwhile, SGLT2 inhibitors are gaining attention for slowing chronic kidney disease progression, supported by European registries confirming RAAS inhibition efficacy in delaying kidney failure.
Alport Syndrome remains a challenging condition with no approved therapies, leading to widespread off-label treatments such as ACE/ARB (used by ~52% of patients), along with statins, allopurinol, antidepressants, and drug–device combinations. Foundations like the National Kidney Foundation and Alport Syndrome Foundation have highlighted the treatment gaps.
There is a strong demand for novel therapies, with key players like Eloxx Pharmaceuticals, Chinook Therapeutics (Novartis), Bayer, Calliditas Therapeutics, and Evotec developing candidates such as ELX-02, Atrasentan, Finerenone, Setanaxib, and BAY3401016. Eloxx’s ELX-02 is anticipated to be the first potential gene therapy approval, followed by Novartis’ Atrasentan, both expected to reshape the treatment paradigm.
Alport Syndrome Market Drivers
- Unmet Medical Need – No FDA/EMA approved therapies, creating strong demand for effective treatment options.
- Emerging Therapies – Pipeline drugs like ELX-02, Atrasentan, Finerenone, Setanaxib, BAY3401016 show promising efficacy and are expected to drive market expansion.
- Increasing Awareness & Advocacy – Patient organizations (e.g., Alport Syndrome Foundation, National Kidney Foundation) are raising disease awareness and pushing for research.
- Rising Prevalence – Growing diagnosed population across the 7MM, with ~160,000 existing cases in 2023, expected to rise by 2034.
- Technological Advancements – Interest in gene therapies and precision medicine to target disease at the molecular level.
- Off-label Therapies as a Bridge – Established use of ACE/ARB, SGLT2 inhibitors, and RAAS inhibitors helps maintain treatment until novel therapies launch.
Alport Syndrome Market Barriers
- Lack of Approved Therapies – Current reliance on off-label treatment (ACE/ARB, statins, SGLT2 inhibitors) limits standardization of care.
- High R&D Costs – Rare disease trials require significant investment with uncertain commercial returns.
- Small Patient Pool – As a rare disease, limited prevalence poses challenges in patient recruitment for clinical trials.
- Regulatory Hurdles – Stringent approval processes and long development timelines for novel therapies.
- Heterogeneity of Disease – Variability in symptoms, progression, and genetic mutations complicates treatment development.
- Limited Real-world Data – Scarcity of long-term observational and prescription studies hinders outcome tracking.
Alport Syndrome Epidemiology
- In 2023, there were about 159,000 prevalent cases of Alport Syndrome across the 7MM (US, EU4, UK, Japan).
- Adults made up ~86% of cases, while pediatrics accounted for ~14%.
- X-linked Alport Syndrome (XLAS) was the most common subtype, with ~11,500 cases in the US, while autosomal dominant (ADAS) was the least common.
- Within EU4, Germany had the highest prevalence (~17,000 cases), while Spain had the lowest.
- In the US, both genders are affected, with slightly higher prevalence in males than females.
Explore more about Alport Syndrome Epidemiology @ Alport Syndrome Market Dynamics and Trends
Alport Syndrome Drugs Uptake
ELX-02 – Eloxx Pharmaceuticals
Eloxx Pharmaceuticals is developing ELX-02, its lead investigational candidate, as a novel small-molecule therapy aimed at restoring the production of full-length, functional proteins. ELX-02 is a synthetic aminoglycoside, administered via subcutaneous and inhalation routes, that enhances ribosomal read-through of premature termination codons (PTCs), thereby enabling the synthesis of functional proteins. It specifically targets the CFTR gene and has shown promising activity in preclinical studies for nonsense mutation–driven genetic kidney disorders. Following encouraging outcomes from its Phase II clinical trial, Eloxx plans to engage with the FDA to finalize the pivotal trial design for ELX-02 in Alport syndrome patients with nonsense mutations. The company is also considering pursuing Breakthrough Therapy Designation. At present, ELX-02 remains in Phase II clinical development for this indication.
Atrasentan – Chinook Therapeutics/Novartis
Atrasentan, developed by Chinook Therapeutics (a Novartis company), is a highly selective endothelin A (ETA) receptor antagonist with potential benefits across several chronic kidney diseases, including IgA nephropathy (IgAN), Alport syndrome, focal segmental glomerulosclerosis (FSGS), and diabetic kidney disease (DKD). The therapy works by reducing proteinuria while exerting anti-inflammatory and anti-fibrotic effects, helping to preserve kidney function.
Originally developed by AbbVie for diabetic kidney disease, the rights to atrasentan were acquired by Chinook in December 2019. Currently, the drug is being evaluated in the Phase II AFFINITY basket trial, which includes four patient cohorts:
- IgAN (with UPCR 0.5 to
- FSGS
- Alport Syndrome
- DKD
Vonafexor – Enyo Pharma
Vonafexor (EYP001), developed by Enyo Pharma, is an innovative therapy under investigation for Alport syndrome and chronic kidney disease (CKD). It is a synthetic, non-steroidal, non-bile acid farnesoid X receptor (FXR) agonist with high selectivity for FXR compared to other nuclear receptors. Importantly, Vonafexor does not interact with the bile acid receptor TGR5. Unlike other FXR agonists, this small molecule has a distinct chemical structure and regulates a unique set of target genes through specific ligand-binding patterns. Currently, Vonafexor is advancing through Phase II clinical development for Alport syndrome.
BAY 3401016 – Bayer
BAY 3401016 (SEMA 3A) is a monoclonal antibody being co-developed by Bayer and Evotec SE. It acts by targeting semaphorin 3A (SEMA3A), a protein that plays a critical role in neuronal guidance, immune regulation, and other biological pathways. The therapy is in the Phase I clinical stage for the treatment of Alport syndrome.
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Alport Syndrome Therapeutics Assessment
Major key companies are working proactively in the Alport Syndrome Therapeutics market to develop novel therapies which will drive the Alport Syndrome treatment markets in the upcoming years are Eloxx Pharmaceuticals (NASDAQ: ELOX), River 3 Renal Corp (NASDAQ: RRRN), Chinook Therapeutics (NASDAQ: KDNY), Travere Therapeutics (NASDAQ: TVTX), Reata Pharmaceuticals (NASDAQ: RETA), and others.
Learn more about the emerging Alport Syndrome therapies & key companies @ Alport Syndrome Clinical Trials and FDA Approvals
Alport Syndrome Report Key Insights
1. Alport Syndrome Patient Population
2. Alport Syndrome Market Size and Trends
3. Key Cross Competition in the Alport Syndrome Market
4. Alport Syndrome Market Dynamics (Key Drivers and Barriers)
5. Alport Syndrome Market Opportunities
6. Alport Syndrome Therapeutic Approaches
7. Alport Syndrome Pipeline Analysis
8. Alport Syndrome Current Treatment Practices/Algorithm
9. Impact of Emerging Therapies on the Alport Syndrome Market
Table of Contents
1. Key Insights
2. Executive Summary
3. Alport Syndrome Competitive Intelligence Analysis
4. Alport Syndrome Market Overview at a Glance
5. Alport Syndrome Disease Background and Overview
6. Alport Syndrome Patient Journey
7. Alport Syndrome Epidemiology and Patient Population
8. Alport Syndrome Treatment Algorithm, Current Treatment, and Medical Practices
9. Alport Syndrome Unmet Needs
10. Key Endpoints of Alport Syndrome Treatment
11. Alport Syndrome Marketed Products
12. Alport Syndrome Emerging Therapies
13. Alport Syndrome Seven Major Market Analysis
14. Attribute Analysis
15. Alport Syndrome Market Outlook (7 major markets)
16. Alport Syndrome Access and Reimbursement Overview
17. KOL Views on the Alport Syndrome Market
18. Alport Syndrome Market Drivers
19. Alport Syndrome Market Barriers
20. Appendix
21. DelveInsight Capabilities
22. Disclaimer
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