Press Release

Alport Syndrome Treatment Market to grow with a CAGR of 4.00%

Increasing healthcare infrastructure and initiatives taken by the government are the major drivers for the Global Alport Syndrome Treatment Market.


According to TechSci Research report, “Global Alport Syndrome Treatment Market Industry Size, Share, Trends, Competition, Opportunity and Forecast, 2018-2028”, Global Alport Syndrome Treatment Market has valued at USD 8.90 million in 2022 and is anticipated to witness an impressive growth in the forecast period with a CAGR of 4.00 % through 2028. This can be ascribed to collaborations and partnerships among leading companies with a diverse approach to merge the expertise of individual companies and to strengthen their position in the market. Advancements in therapeutic approaches in Alport syndrome treatment are critical in addressing the unmet medical needs of affected individuals. Advances in genetic research have paved the way for precision medicine in Alport syndrome treatment. Identifying specific genetic mutations allows for personalized treatment approaches that target the underlying genetic cause of the disease. This approach aims to develop therapies tailored to an individual's genetic subtype. RNA-targeted genetic therapies, such as antisense oligonucleotides (ASOs) and small interfering RNA (siRNA), are being investigated as potential treatments for Alport syndrome.

These therapies aim to modify or correct the expression of mutated genes responsible for the condition. Exosomes, tiny vesicles secreted by cells, have shown potential in delivering therapeutic molecules to target tissues. Researchers are exploring exosome-based therapies to deliver genetic material or therapeutic agents to affected kidney cells. The identification of specific biomarkers associated with Alport syndrome can aid in early diagnosis and treatment monitoring. Biomarker development is an active area of research in the field. Inflammation plays a role in the progression of kidney damage in Alport syndrome. Emerging therapies include the use of anti-inflammatory agents to reduce kidney inflammation and slow disease progression.

Alport syndrome is a rare genetic disorder characterized by kidney disease, hearing impairment, and vision issues. It results from mutations in the COL4A3, COL4A4, or COL4A5 genes, which are responsible for producing crucial proteins like type IV Collagen. Individuals with Alport syndrome experience a gradual decline in kidney function, often indicated by the presence of blood in the urine as a sign of impaired kidney function. Many Alport syndrome patients also exhibit elevated protein levels in their urine. With disease progression, kidney function deteriorates, ultimately leading to End-Stage Renal Disease (ESRD). Furthermore, Alport syndrome is associated with inner ear abnormalities, resulting in sensorineural hearing loss, as well as vision impairment.

In February 2023, Eloxx Pharmaceuticals, Inc., a pioneer in the development of genetic therapies focused on ribosomal RNA, has commenced the dosing of the initial patients in its Phase 2 clinical trial for ELX-02. This trial aims to assess the potential of ELX-02 as a treatment for Alport syndrome in patients with nonsense mutations. In this Phase 2 trial, the focus is on administering ELX-02 to a maximum of eight Alport syndrome patients who possess nonsense mutations in the COL4 gene. These patients will undergo a two-month dosing regimen, followed by a three-month monitoring period. In addition to prioritizing safety as the primary endpoint, the study will also closely monitor a significant secondary efficacy endpoint, which is the measurement of proteinuria every two weeks. Proteinuria is a well-established endpoint used in evaluating treatments for various kidney-related conditions and serves as a reliable predictor of treatment effectiveness. Furthermore, for eligible participants, the induction of COL4 will be assessed at the conclusion of the two-month period.


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Limited treatment options pose a significant challenge in the global Alport syndrome treatment market. There are currently no disease-modifying treatments available for Alport syndrome. This means that existing therapies primarily focus on symptom management and slowing disease progression rather than addressing the underlying genetic cause of the condition. The available treatments for Alport syndrome, such as blood pressure control and medications to reduce proteinuria, have limited efficacy in preventing kidney disease progression. While they can be beneficial, they may not halt the disease's advance in many cases. Treatment options for hearing loss associated with Alport syndrome are often limited to hearing aids or cochlear implants. These devices can improve hearing to some extent but may not fully restore normal auditory function. Alport syndrome patients and their families often seek curative therapies that can address the genetic mutations responsible for the disease. The absence of curative options can be frustrating and disappointing. Advances in genetic research have raised hopes for personalized medicine approaches to treat Alport syndrome. Limited treatment options hinder the development of targeted therapies tailored to an individual's specific genetic subtype.

Global Alport Syndrome Treatment Market segmentation is based on Product Type, Disease Type, End User, By Company, and Region.

Based on Disease Type, Global Alport Syndrome Treatment Market is segmented into X-linked Alport Syndrome, Autosomal Recessive Alport Syndrome, Autosomal Dominant Alport Syndrome. Autosomal recessive Alport syndrome is one of the genetic inheritance patterns of Alport syndrome, a rare genetic disorder that primarily affects the kidneys, ears, and eyes. In autosomal recessive Alport syndrome, the condition is caused by mutations in specific genes, and it follows an inheritance pattern that can affect both males and females. Autosomal recessive Alport syndrome is inherited when an individual inherits two mutated copies of the relevant genes, one from each parent. These mutated genes are typically located on autosomal chromosomes, which are non-sex chromosomes (i.e., not the X or Y chromosomes). The progression of autosomal recessive Alport syndrome can vary among individuals. It often leads to kidney insufficiency or failure over time, necessitating kidney replacement therapies such as dialysis or transplantation.

Based on Region, the North America segment is the dominating segment of Global Alport Syndrome Treatment Market. North America is home to numerous pharmaceutical and biotechnology companies with a focus on rare diseases. These companies play a crucial role in developing and commercializing treatments for Alport syndrome. The region's relatively large population allows for a sufficient pool of patients for clinical trials and the establishment of specialized clinics and centers of excellence for rare diseases. In countries like the United States and Canada, health insurance coverage may help facilitate access to costly treatments, reducing the financial burden on patients and their families. North American researchers and organizations often collaborate with international counterparts, contributing to a global effort to advance Alport syndrome research and treatment. While healthcare access varies within North America, the region generally provides relatively good access to healthcare services, diagnostics, and specialized care for rare diseases.

Asia Pacific is the fastest-growing region in the Global Alport Syndrome Treatment Market. Awareness about rare diseases like Alport syndrome is growing in the Asia Pacific region. As healthcare providers and patients become more informed, there is greater recognition and diagnosis of the condition, leading to increased demand for treatment. Many countries in the Asia Pacific have been investing in expanding their healthcare infrastructure. This includes the establishment of specialized centers and clinics for rare diseases, improving access to diagnosis, treatment, and management of Alport syndrome. Advances in genetic testing have made it easier to diagnose Alport syndrome and identify specific genetic mutations. Improved access to genetic testing facilities in the Asia Pacific has contributed to early diagnosis and targeted treatment. The region has witnessed a growing interest in rare disease research, including Alport syndrome. Pharmaceutical companies and research institutions are conducting clinical trials and studies in the Asia Pacific, making novel treatments and therapies more accessible.


Some of the major companies operating in the Global Alport Syndrome Treatment Market include:

  • Retrophin, Inc.
  • Goldfinch Bio, Inc.
  • Regulus Therapeutics Inc.
  • Vertex Pharmaceuticals Incorporated
  • ChemoCentryx, Inc.
  • Reata Pharmaceuticals, Inc.
  • Sanofi S.A.
  • Ionis Pharmaceuticals, Inc.
  • Omeros Corporation
  • Akebia Therapeutics, Inc.


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“Certain areas, particularly in North America, are projected to exert significant demand of Alport Syndrome Treatment. The growth in the competitive landscape and the presence of well-established companies in the market, committed to enhance the overall wellbeing of people each year, are expected to contribute to a remarkable growth of the Global Alport Syndrome Treatment Market in the forecast period," said Mr. Karan Chechi, Research Director with TechSci Research, a research-based Global management consulting firm.

Alport Syndrome Treatment Market - Global Industry Size, Share, Trends, Opportunity, and Forecast, 2018-2028  Segmented by Product Type (Tablets, Capsules, Injectable, Others), By Disease Type (X-linked Alport Syndrome, Autosomal Recessive Alport Syndrome, Autosomal Dominant Alport Syndrome), By End User (Hospital Pharmacies, Retail Pharmacies, Specialty Clinics), By Region, and Competition has evaluated the future growth potential of Global Alport Syndrome Treatment Market and provides statistics & information on market size, structure, and future market growth. The report intends to provide innovative market intelligence and help decision makers take sound investment decisions. Besides, the report also identifies and analyzes the emerging trends along with essential drivers, challenges, and opportunities in Global Alport Syndrome Treatment Market.



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