Glutaric Acidemia Type 1 Market

Market Overview:

The 7 major glutaric acidemia type 1 markets reached a value of USD 530.5 Million in 2024. Looking forward, IMARC Group expects the 7MM to reach USD 947.4 Million by 2035, exhibiting a growth rate (CAGR) of 5.43% during 2025-2035.

The glutaric acidemia type 1 market has been comprehensively analyzed in IMARC's new report titled "Glutaric Acidemia Type 1 Market: Epidemiology, Industry Trends, Share, Size, Growth, Opportunity, and Forecast 2025-2035". Glutaric acidemia type 1 (GA-1) is an autosomal recessive condition indicated by a lack of glutaryl-CoA dehydrogenase (GCDH) activity. This causes glutaric acid to accumulate in tissues and be excreted in the urine of affected patients. GCD is involved in the breakdown of the amino acids lysine, hydroxylysine, and tryptophan. The severity of GA-1 varies significantly; some people are just minimally affected, while others experience serious problems. Signs and symptoms typically appear in infancy or early childhood; however, in a small number of cases, the condition manifests itself in adolescence or adulthood. Many babies born with this disease have abnormally large heads (macrocephaly). Affected people may also have difficulties moving and have spasms, jerks, rigidity, or decreased muscular tone. Some GA-1 patients have experienced bleeding in the brain or eyes, which could be misinterpreted as the consequence of child abuse. The illness is primarily diagnosed through neonatal blood spot screening, which identifies high levels of glutaric acid and 3-hydroxyglutaric acid in the urine. The healthcare provider also recommends additional studies, including enzyme examination of cultured fibroblasts, genetic testing for GCDH gene alterations, and screening of blood and cerebrospinal fluid for 3-hydroxyglutaric acid.

The escalating prevalence of mutations in the GCDH gene, which provides instructions for making the enzyme glutaryl-CoA dehydrogenase, is primarily driving the glutaric acidemia type 1 market. In addition to this, the inflating utilization of carnitine supplementation to help in minimizing the accumulation of toxic glutaric acid in the body and improving the outcomes for patients is also creating a positive outlook for the market. Moreover, the rising usage of genetic testing using next-generation sequencing and whole-exome sequencing that can identify mutations in the GCDH gene for definitive diagnosis of GA-1 in individuals with atypical biochemical profiles or late-onset symptoms is further bolstering the market growth. Apart from this, the widespread adoption of dietary therapy, which primarily involves a low-protein diet, specifically restricting the intake of lysine and tryptophan to reduce toxic metabolite buildup and prevent neurological complications, is acting as another significant growth-inducing factor. Additionally, the emerging popularity of gene therapy, since it delivers a functional copy of the GCDH gene, thereby effectively replacing the defective enzyme and mitigating the disease symptoms, is expected to drive the glutaric acidemia type 1 market during the forecast period.

IMARC Group's new report provides an exhaustive analysis of the glutaric acidemia type 1 market in the United States, EU5 (Germany, Spain, Italy, France, and 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 glutaric acidemia type 1 and also represents the largest market for its treatment. Furthermore, the current treatment practice/algorithm, market drivers, challenges, opportunities, reimbursement scenario and 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 glutaric acidemia type 1 market in any manner.

Drugs:

VGM-R02b is an investigational gene replacement therapy designed to address glutaric acidemia type I. The mechanism of action of VGM-R02b involves delivering a functional copy of the GCDH gene to patients' cells, thereby restoring the production of active glutaryl-CoA dehydrogenase enzyme. This restoration enables the proper metabolism of lysine, hydroxylysine, and tryptophan, preventing the accumulation of harmful metabolites and potentially improving clinical outcomes for affected individuals.

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 glutaric acidemia type 1 market
• Historical, current, and future performance of various therapeutic categories in the market
• Sales of various drugs across the glutaric acidemia type 1 market
• Reimbursement scenario in the market
• In-market and pipeline drugs

Competitive Landscape:

This report also provides a detailed analysis of the current glutaric acidemia type 1 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 glutaric acidemia type 1 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 glutaric acidemia type 1 market across the seven major markets in 2024 and what will it look like in 2035?
• What is the growth rate of the glutaric acidemia type 1 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 glutaric acidemia type 1 across the seven major markets?
• What is the number of prevalent cases (2019-2035) of glutaric acidemia type 1 by age across the seven major markets?
• What is the number of prevalent cases (2019-2035) of glutaric acidemia type 1 by gender across the seven major markets?
• How many patients are diagnosed (2019-2035) with glutaric acidemia type 1 across the seven major markets?
• What is the size of the glutaric acidemia type 1 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 glutaric acidemia type 1?
• What will be the growth rate of patients across the seven major markets?

Glutaric Acidemia Type 1: 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 glutaric acidemia type 1 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 glutaric acidemia type 1 market?
• What are the key regulatory events related to the glutaric acidemia type 1 market?
• What is the structure of clinical trial landscape by status related to the glutaric acidemia type 1 market?
• What is the structure of clinical trial landscape by phase related to the glutaric acidemia type 1 market?
• What is the structure of clinical trial landscape by route of administration related to the glutaric acidemia type 1 market?