Global Choroideremia Treatment Market (2024 Edition): Market Size, Trends, Opportunities and Forecast by Treatment Type (Gene Therapy, Topical Carbonic Inhibitor, Dorzolamide, Lutein and Luxturna), Distribution Channel, Route of Administration, Region, By

Global Choroideremia Treatment Market (2024 Edition): Market Size, Trends, Opportunities and Forecast by Treatment Type (Gene Therapy, Topical Carbonic Inhibitor, Dorzolamide, Lutein and Luxturna), Distribution Channel, Route of Administration, Region, By Country: 2020-2030

Background

A report published by Azoth Analytics highlights different factors and opportunities prevailing in the “Global Choroideremia Treatment Market (2024 Edition)”. According to the report, Global Choroideremia Treatment Market valued at USD 9.33 Billion in 2023 is expected to grow at a CAGR of 6.2% during 2025-2030.

The report explores a multi-faceted opportunity landscape in terms of market segmentation by Treatment Type (Gene Therapy, Topical Carbonic Inhibitor, Dorzolamide, Lutein and Luxturna), by Distribution Channel (Online Pharmacies, Hospital Pharmacies, Retail Pharmacies and Drugstores) and by Route of Administration (Oral & Intravenous and Ophthalmic)

Market Overview

The Choroideremia treatment market is experiencing significant growth, driven by advancements in medical research and an increasing understanding of this rare genetic disorder. Choroideremia, an inherited retinal disease primarily affecting males, leads to progressive vision loss and eventual blindness due to the degeneration of the choroid, retinal pigment epithelium (RPE), and photoreceptors. The market's expansion is fueled by the development of innovative therapies, particularly gene therapies, which offer promising prospects for altering the disease's progression and improving patients' quality of life.

Choroideremia is caused by mutations in the CHM gene, which encodes the Rab escort protein-1 (REP-1). This protein is essential for intracellular trafficking, and its absence disrupts cellular functions, leading to cell death in the retinal layers. The disorder's pathophysiology and genetic basis have been well-characterized, paving the way for targeted therapeutic interventions. The primary aim of current treatment strategies is to halt or slow down the degeneration process, thereby preserving vision for as long as possible.

Gene therapy has emerged as a revolutionary approach in the Choroideremia treatment market. This technique involves delivering a functional copy of the CHM gene to the affected cells in the retina. By doing so, it aims to restore the production of REP-1 protein, thereby preventing further degeneration. One of the most advanced candidates in this domain is the gene therapy product BIIB111 (formerly known as NSR-REP1) developed by Biogen. Clinical trials have shown that BIIB111 can be safely administered and has the potential to stabilize or improve visual function in patients with Choroideremia.

Another notable player in the gene therapy space is 4D Molecular Therapeutics, which is developing 4D-110, a gene therapy designed to deliver the CHM gene using a novel adeno-associated virus (AAV) vector. This therapy is currently undergoing clinical trials, and early results have demonstrated its safety and efficacy in delivering the therapeutic gene to the retina. The use of AAV vectors is particularly advantageous due to their ability to target retinal cells effectively and their established safety profile.

Apart from gene therapy, other therapeutic approaches are also being explored. For instance, retinal implants and prosthetics, like those developed by Retina Implant AG, offer an alternative for patients in advanced stages of the disease. These devices can potentially restore partial vision by electronically stimulating the remaining functional retinal cells.

Although still in the experimental stage, these technologies represent a crucial area of research and development in the Choroideremia treatment market.

The market dynamics are also influenced by several key players investing in research and development. Companies such as Novartis AG, Spark Therapeutics, Inc., bluebird bio, Inc., and Copernicus Therapeutics, Inc. are at the forefront of these efforts. Their extensive R&D pipelines include various gene therapy candidates at different stages of clinical trials. Novartis AG, for instance, has been investing heavily in gene therapy research, leveraging its expertise in ophthalmology to develop novel treatments for retinal diseases, including Choroideremia.

In addition to corporate investments, academic and research institutions play a pivotal role in advancing the understanding of Choroideremia and developing new treatments. Collaborations between pharmaceutical companies and academic researchers are common, facilitating the translation of basic research findings into clinical applications. For example, ongoing clinical trials are often conducted in partnership with leading medical institutions, ensuring rigorous evaluation of new therapies.

The regulatory landscape for Choroideremia treatments is another critical factor shaping the market. Regulatory bodies such as the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) have established frameworks for the approval of gene therapies, which are categorized as advanced therapy medicinal products (ATMPs). These frameworks ensure that new treatments meet stringent safety and efficacy standards before they can be marketed. The approval of Luxturna (voretigene neparvovec-rzyl), a gene therapy for another inherited retinal disease, serves as a precedent and has paved the way for similar therapies targeting Choroideremia.

Patient advocacy groups also significantly impact the market by raising awareness about Choroideremia, funding research, and advocating for better access to treatments. Organizations such as the Choroideremia Research Foundation (CRF) and Fight for Sight provide essential support for research initiatives and clinical trials. Their efforts help bridge the gap between researchers, clinicians, and patients, fostering a collaborative environment that accelerates the development of effective therapies.

Segment Insights

By Distribution Channel, Hospital Pharmacies segment contributes the maximum revenue with a share of approximately 41.3% in 2023.

Gene therapy, a burgeoning area in the treatment of Choroideremia, involves the introduction of a functional copy of the CHM gene into retinal cells to compensate for the defective gene causing the disease. Hospital pharmacies play a pivotal role in this context by managing the logistics of gene therapy products, which often require stringent storage and handling conditions to maintain their efficacy. For instance, therapies like BIIB111 (developed by Biogen) and 4D-110 (developed by 4D Molecular Therapeutics) are advanced treatments that necessitate the sophisticated capabilities of hospital pharmacies for proper administration.

Geographical Insights

Americas represents the largest market in value terms, by region with a market share of 36.3% in the Global Choroideremia Treatment Market in 2023.

The Choroideremia treatment market in the Americas is characterized by significant advancements in medical research, robust healthcare infrastructure, and substantial investments in developing innovative therapies. Choroideremia, a rare inherited retinal disorder primarily affecting males, leads to progressive vision loss and eventual blindness due to the degeneration of the choroid, retinal pigment epithelium (RPE), and photoreceptors. This market's growth is driven by the increasing prevalence of the disease, the advancement of gene therapies, and supportive regulatory frameworks that facilitate the development and approval of new treatments.

In North America, the United States stands at the forefront of the Choroideremia treatment market. The country's leading position is attributed to its advanced healthcare system, high healthcare expenditure, and the presence of numerous pharmaceutical and biotechnology companies actively involved in research and development. Companies like Biogen, 4D Molecular Therapeutics, and Spark Therapeutics have been pivotal in developing gene therapies aimed at treating Choroideremia. Biogen's gene therapy candidate BIIB111 (formerly known as NSR-REP1) has shown promising results in clinical trials, demonstrating its potential to stabilize or improve visual function in patients with Choroideremia. Similarly, 4D Molecular Therapeutics is developing 4D-110, a gene therapy that uses a novel adeno-associated virus (AAV) vector to deliver the CHM gene to retinal cells, which has also shown encouraging early results.

Market Segmentation

By Treatment Type:

• Gene Therapy
• Topical Carbonic Inhibitor
• Dorzolamide
• Lutein
• Luxturna

By Distribution Channel:

• Online Pharmacies
• Hospital Pharmacies
• Retail Pharmacies
• Drugstores

By Route of Administration:

• Oral & Intravenous
• Ophthalmic

By Geography

• Americas (The United States, Canada, Brazil, Mexico, Rest of Americas)
• Europe (United Kingdom, Germany, France, Italy, Spain, Turkey, Russia, Rest of Europe)
• Asia Pacific (China, Japan, South Korea, India, Singapore, Indonesia, Rest of Asia Pacific)
• Middle East and Africa (GCC, Israel, South Africa, Rest of Middle East and Africa)

Key Companies

The report assesses the competitive developments, strategies, mergers and acquisitions and new product development and market share portfolio of top 15 companies. Key companies in the Global Choroideremia Treatment Market include:

• F. Hoffmann-La Roche Ltd
• Retina Implant AG
• 4D Molecular Therapeutics
• Biogen
• Spark Therapeutics, Inc.
• Copernicus Therapeutics, Inc.
• Novartis AG
• bluebird bio, Inc.
• Wize Pharma Inc.
• Pixium Vision
• Other Leading Companies

Reasons to buy this report:

• In-Depth Comparative Assessment of Top 20 Markets in the Choroideremia Treatment Industry: Comparative assessment of 20 leading countries highlighting the total addressable market, opportunities, lucrative segments and competitive positioning of leading companies.
• Comprehensive Historical, Present, and Future Analytics of the Choroideremia Treatment Market: A deep dive in the historical (2020-2023), current (2024) and forecast (2025-2030) market analytics of Choroideremia Treatment Market.
• Detailed Analysis of Choroideremia Treatment Market By Treatment Type, Distribution Channel, and Route of Administration Across 20 Countries
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