The 7 major canavan disease markets reached a value of USD 260.6 Million in 2024. Looking forward, IMARC Group expects the 7MM to reach USD 397.3 Million by 2035, exhibiting a growth rate (CAGR) of 3.91% during 2025-2035.
Report Attribute
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Key Statistics
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Base Year | 2024 |
Forecast Years | 2025-2035 |
Historical Years |
2019-2024
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Market Size in 2024
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USD 260.6 Million |
Market Forecast in 2035
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USD 397.3 Million |
Market Growth Rate (2025-2035)
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3.91% |
The canavan disease market has been comprehensively analyzed in IMARC's new report titled "Canavan Disease Market: Epidemiology, Industry Trends, Share, Size, Growth, Opportunity, and Forecast 2025-2035". Canavan disease is a severe, lethal inherited disorder that is primarily associated with the central nervous system, resulting in progressive neurological decline. It is a type of leukodystrophy, a category of conditions that involve the degeneration of white matter in the brain as a result of abnormal myelin development. Canavan disease is caused by mutations in the ASPA gene, which codes for the enzyme aspartoacylase. This enzyme catalyzes the degradation of N-acetylaspartic acid (NAA), a metabolite important in brain cell metabolism. A lack or deficiency of aspartoacylase leads to toxic NAA accumulation, causing disordered myelin formation, thus severe brain dysfunction. The disorder appears during infancy, and these children have manifestations of hypotonia (hypotonia = low muscle tone), macrocephaly (abnormally large head), weakness in motor functions, and delays in development. Symptoms include seizures, loss of sight, dysphagia, and inability to make voluntary movements as the illness advances. Genetic tests, enzyme activities, and innovative imaging such as magnetic resonance spectroscopy (MRS) diagnose Canavan disease by showing levels of high NAA in the brain.
The growing realization of genetic diseases, developments in newborn screening programs, and growing numbers of verified cases are principal forces driving the Canavan disease market. Also contributing is the development of gene therapy, for example, investigational drugs aiming to target ASPA mutations in order to revive enzymatic function, that are significantly driving the market's growth. The emergence of adeno-associated virus (AAV)-based gene therapies, such as investigative candidates like ASPA gene replacement therapy, is also driving research and investment in the field. In addition, supportive therapies such as physical therapy, speech therapy, and nutritional support continue to play a key role in disease management, and the increasing need for multidisciplinary care strategies is evident. The Canavan disease market is seeing a rise in clinical testing, with several gene therapy compounds progressing through preclinical and initial-phase studies. Research institutions and companies are investigating new therapeutic strategies, including enzyme replacement therapy and substrate reduction therapy, to slow the disease progression. Increased emphasis on developing drugs for rare diseases, combined with regulatory incentives like orphan drug designations and fast-track designation, is fueling market growth.
IMARC Group's new report provides an exhaustive analysis of the canavan disease market in the United States, EU4 (Germany, Spain, Italy, and France), United Kingdom, and Japan. This includes treatment practices, in-market, and pipeline drugs, share of individual therapies, market performance across the seven major markets, market performance of key companies and their drugs, etc. The report also provides the current and future patient pool across the seven major markets. According to the report, the United States has the largest patient pool for canavan disease and also represents the largest market for its treatment. Furthermore, the current treatment practice/algorithm, market drivers, challenges, opportunities, reimbursement scenario, unmet medical needs, etc., have also been provided in the report. This report is a must-read for manufacturers, investors, business strategists, researchers, consultants, and all those who have any kind of stake or are planning to foray into the canavan disease market in any manner.
BBP-812 is a clinical trial AAV9 gene therapy designed to restore the function of the ASPA gene in Canavan disease. Preclinical data indicate enhanced survival and motor function. The treatment is FDA Fast Track, Rare Pediatric Disease, and Orphan Drug designated, noting its promise for treating this orphan neurodegenerative disorder.
CP-102, created by Contera Pharma, is an experimental treatment for Canavan disease. It aims at the underlying metabolic impairment related to ASPA gene mutations. Formulated to regulate disease advancement, CP-102 is expected to enhance neurological outcomes through toxic metabolite accumulation reduction, presenting a potential treatment for this orphan neurodegenerative disease.
Myrtelle's rAAV-Olig001-ASPA is a gene therapy aimed at treating Canavan disease by treating oligodendrocytes. It transduces a functional ASPA gene to improve the production of myelin to restore motor and cognitive function. The treatment is designated as an Orphan Drug, Fast-Track, and Rare Pediatric Disease in the U.S. and Orphan Drug in Europe.
Time Period of the Study
Countries Covered
Analysis Covered Across Each Country
This report also provides a detailed analysis of the current canavan disease marketed drugs and late-stage pipeline drugs.
In-Market Drugs
Late-Stage Pipeline Drugs
Drugs | Company Name |
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BBP 812 | Aspa Therapeutics |
CP 102 | Contera Pharma |
rAAV-Olig001 ASPA | Myrtelle |
*Kindly note that the drugs in the above table only represent a partial list of marketed/pipeline drugs, and the complete list has been provided in the report.
Market Insights
Epidemiology Insights
Canavan Disease: Current Treatment Scenario, Marketed Drugs and Emerging Therapies