Canavan Disease Market

Market Overview:

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.

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.

Recent Developments:

• In April 2024, Pfizer Inc. and Genmab A/S announced that the U.S. Food and Drug Administration (FDA) had approved the supplemental Biologics License Application (sBLA) for TIVDAK (tisotumab vedotin-tftv) for the treatment of recurrent or metastatic canavan disease with disease progression during or after chemotherapy.
• In June 2024, the FDA chose Myrtelle's gene therapy for inclusion in its START Pilot Program, with the intention of speeding up Canavan disease treatment development by enhancing regulatory interaction and accelerating approval.

Key Highlights:

• The disease is rare worldwide but has a higher occurrence among individuals of Ashkenazi Jewish descent, with an estimated frequency ranging from 1 in 6,400 to 13,500 in this population.
• Around 1 in 40 Ashkenazi Jews carry the ASPA gene mutation, increasing the risk of Canavan disease in this group.
• Symptoms appear between 3-9 months, including poor muscle tone, enlarged head, head control issues, and developmental delays.
• Many affected individuals do not survive beyond childhood, though some may live into adolescence or early adulthood.
• Mutations in the ASPA gene cause aspartoacylase deficiency, leading to NAA accumulation in the brain, impairing neurological development.

Drugs:

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

• Base Year: 2024
• Historical Period: 2019-2024
• Market Forecast: 2025-2035

Countries Covered

• United States
• Germany
• France
• United Kingdom
• Italy
• Spain
• Japan

Analysis Covered Across Each Country

• Historical, current, and future epidemiology scenario
• Historical, current, and future performance of the canavan disease market
• Historical, current, and future performance of various therapeutic categories in the market
• Sales of various drugs across the canavan disease market
• Reimbursement scenario in the market
• In-market and pipeline drugs

Competitive Landscape:

This report also provides a detailed analysis of the current canavan disease marketed drugs and late-stage pipeline drugs.

In-Market Drugs

• Drug Overview
• Mechanism of Action
• Regulatory Status
• Clinical Trial Results
• Drug Uptake and Market Performance

Late-Stage Pipeline Drugs

• Drug Overview
• Mechanism of Action
• Regulatory Status
• Clinical Trial Results
• Drug Uptake and Market Performance

*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.

Key Questions Answered in this Report:

Market Insights

• How has the canavan disease market performed so far and how will it perform in the coming years?
• What are the markets shares of various therapeutic segments in 2024 and how are they expected to perform till 2035?
• What was the country-wise size of the canavan disease market across the seven major markets in 2024 and what will it look like in 2035?
• What is the growth rate of the canavan disease market across the seven major markets and what will be the expected growth over the next ten years?
• What are the key unmet needs in the market?

Epidemiology Insights

• What is the number of prevalent cases (2019-2035) of canavan disease across the seven major markets?
• What is the number of prevalent cases (2019-2035​) of canavan disease by age across the seven major markets?
• What is the number of prevalent cases (​2019-2035​) of canavan disease by gender across the seven major markets?
• How many patients are diagnosed (​2019-2035​) with canavan disease across the seven major markets?
• What is the size of the canavan disease patient pool (2019-2024) across the seven major markets?
• What would be the forecasted patient pool (2025-2035) across the seven major markets?
• What are the key factors driving the epidemiological trend of canavan disease?
• What will be the growth rate of patients across the seven major markets?

Canavan Disease: Current Treatment Scenario, Marketed Drugs and Emerging Therapies

• What are the current marketed drugs and what are their market performance?
• What are the key pipeline drugs and how are they expected to perform in the coming years?
• How safe are the current marketed drugs and what are their efficacies?
• How safe are the late-stage pipeline drugs and what are their efficacies?
• What are the current treatment guidelines for canavan disease drugs across the seven major markets?
• Who are the key companies in the market and what are their market shares?
• What are the key mergers and acquisitions, licensing activities, collaborations, etc. related to the canavan disease market?
• What are the key regulatory events related to the canavan disease market?
• What is the structure of clinical trial landscape by status related to the canavan disease market?
• What is the structure of clinical trial landscape by phase related to the canavan disease market?
• What is the structure of clinical trial landscape by route of administration related to the canavan disease market?