DiGeorge Syndrome Market

Market Overview:

The 7 major diGeorge syndrome markets are expected to exhibit a CAGR of 4.78% during 2024-2034.

The DiGeorge Syndrome market has been comprehensively analyzed in IMARC's new report titled "DiGeorge Syndrome Market: Epidemiology, Industry Trends, Share, Size, Growth, Opportunity, and Forecast 2024-2034". DiGeorge syndrome, also called 22q11.2 deletion syndrome, refers to a genetic disorder caused by a small piece of chromosome 22 being missing. This disorder presents a wide range of symptoms that can vary in severity, affecting multiple systems of the body. Common indications include congenital heart defects, cleft palate, immune system dysfunction, developmental delays, learning difficulties, and characteristic facial features. Additionally, individuals suffering from DiGeorge Syndrome might experience issues related to the thymus and parathyroid glands, leading to problems with calcium regulation and susceptibility to infections. The diagnosis of the illness typically involves a combination of clinical evaluation, medical history assessment, and genetic testing to identify the missing piece of chromosome 22. Prenatal testing through techniques like amniocentesis can be used to diagnose the syndrome before birth if there is a suspicion due to family history or specific ultrasound findings. Early detection is crucial for managing the diverse array of symptoms associated with the disease.

The escalating prevalence of chromosomal microdeletions in the 22q11.2 region that can affect the thymus and parathyroid glands is primarily driving the DiGeorge Syndrome market. In addition to this, the inflating utilization of pharmacological agents, such as immunoglobulin therapies for boosting the immune system and calcium supplements for managing hypocalcemia in individuals suffering from the ailment, is also creating a positive outlook for the market. Moreover, the widespread adoption of specialized care services, like speech and occupational therapy, that helps to improve the quality of life for patients by enhancing speech clarity and motor skills is further bolstering the market growth. Apart from this, the rising usage of gene editing techniques, including CRISPR-Cas9, since it holds promise for directly correcting the genetic deletion responsible for DiGeorge Syndrome is acting as another significant growth-inducing factor. Additionally, the emerging popularity of cardiac interventions to address congenital heart defects often associated with the condition is also augmenting the market growth. Furthermore, numerous advancements in diagnostic methodologies, like fluorescence in situ hybridization (FISH) and array comparative genomic hybridization (aCGH), which aid in early and accurate diagnosis, thereby amplifying the prospects for targeted treatments, are expected to drive the DiGeorge Syndrome market during the forecast period.

IMARC Group's new report provides an exhaustive analysis of the DiGeorge Syndrome 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 DiGeorge Syndrome 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 DiGeorge Syndrome market in any manner.

Recent Developments:

• In October 2023, Nobias Therapeutics unveiled top-line data from a Phase 2 clinical trial investigating NB-001 (Fasoracetam) as a potential treatment for the neuropsychiatric symptoms associated with 22q11.2 deletion syndrome in children. The trial reached its primary endpoint by demonstrating the safety and tolerability of NB-001, while efficacy measures provide support for its progression to a registrational trial.

Key Highlights:

• The DiGeorge Syndrome impacts approximately 0.1% of fetuses. In live births, it is estimated to occur at a rate of 1 in 4000 to 6000. Various factors contribute to the variation in prevalence between fetuses and live births.
• The frequency of DGS is roughly one in 3,000-6,000 individuals and exhibits an ongoing annual rise. Gender or race does not present significant risk factors, as both males and females of any ethnic background are equally prone to the condition.
• The annual prevalence rate of DiGeorge Syndrome in the United States is approximately 1 in 4,000 people.

Drugs:

RVT-802 is the only drug approved by the European Commission for the treatment of DiGeorge Syndrome, NB-001(Fasoracetam), Zygel (ZYN002 Cannabidiol Gel) and others are drug molecules in the clinical stage for DiGeorge Syndrome.

RVT-802, also known as allogeneic cultured postnatal thymus-derived tissue. It is designed as a treatment for congenital athymia, specifically focusing on DiGeorge Syndrome cases. Enzyvant Therapeutics Ireland Limited, situated in Ireland, has received authorization from the European Commission for the application of RVT-802 in the treatment of DiGeorge syndrome.

NB-001 (Fasoracetam) is a pipeline drug, that functions as a non-stimulant modulator of metabotropic glutamate receptors, targeting metabotropic glutamate receptors 1, 3, 5, 7, 8, as well as GABAB receptors. Fasoracetam monohydrate is currently in development as a therapeutic option for addressing DiGeorge Syndrome (22q11.2 deletion syndrome) and unspecified lymphatic anomalies. The drug is currently available in phase 2 clinical trials.

Zygel (ZYN002 Cannabidiol Gel) is a pipeline drug in development, distinguished as the initial and sole pharmaceutical-grade cannabidiol preparation. This non-psychoactive cannabinoid is structured as a patented permeation-enhanced gel for transdermal administration, facilitating absorption through the skin and into the bloodstream. Zygel is undergoing clinical trials in Phase 2 for patients with Fragile X syndrome (FXS) and 22q11.2 deletion syndrome (22q).

Time Period of the Study

• Base Year: 2023
• Historical Period: 2018-2023
• Market Forecast: 2024-2034
   

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

Competitive Landscape:

This report also provides a detailed analysis of the current DiGeorge syndrome 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 DiGeorge syndrome market performed so far and how will it perform in the coming years?
• What are the markets shares of various therapeutic segments in 2023 and how are they expected to perform till 2034?
• What was the country-wise size of the DiGeorge syndrome market across the seven major markets in 2023 and what will it look like in 2034?
• What is the growth rate of the DiGeorge syndrome 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 (2018-2034) of DiGeorge syndrome across the seven major markets?
• What is the number of prevalent cases (2018-2034) of DiGeorge syndrome by age across the seven major markets?
• What is the number of prevalent cases (2018-2034) of DiGeorge syndrome by gender across the seven major markets?
• How many patients are diagnosed (2018-2034) with DiGeorge syndrome across the seven major markets?
• What is the size of the DiGeorge syndrome patient pool (2018-2023) across the seven major markets?
• What would be the forecasted patient pool (2024-2034) across the seven major markets?
• What are the key factors driving the epidemiological trend of DiGeorge syndrome?
• What will be the growth rate of patients across the seven major markets?
   

DiGeorge Syndrome: 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 DiGeorge syndrome 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 DiGeorge syndrome market?
• What are the key regulatory events related to the DiGeorge syndrome market?
• What is the structure of clinical trial landscape by status related to the DiGeorge syndrome market?
• What is the structure of clinical trial landscape by phase related to the DiGeorge syndrome market?
• What is the structure of clinical trial landscape by route of administration related to the DiGeorge syndrome market?