DelveInsight’s “Angelman Syndrome Market Insights, Epidemiology, and Market Forecast 2032” report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the Angelman Syndrome market size, share, trends, and growth opportunities in the seven major markets (7MM) (i.e., the United States, EU4 (Germany, Spain, Italy, France), the United Kingdom and Japan).
The report covers emerging Angelman Syndrome drugs, current treatment practices, market share of individual therapies, and current & forecasted market size from 2019 to 2032. It also evaluates the current Angelman Syndrome treatment practice/algorithm, key drivers & barriers impacting the market growth, and unmet medical needs to curate the best of the opportunities and assess the underlying potential of the market.
Angelman Syndrome: An Overview
According to the National Organization for Rare Disorders, Angelman syndrome is a rare genetic and neurological disorder characterized by severe developmental delay and learning disabilities; absence or near absence of speech; inability to coordinate voluntary movements (ataxia); tremulousness with jerky movements of the arms and legs and a distinct behavioral pattern characterized by a happy disposition and unprovoked episodes of laughter and smiling.
People with Angelman syndrome have developmental problems that become noticeable by the age of 6 – 12 months. Other common signs and symptoms usually appear in early childhood like walking and balance disorders, gastrointestinal issues, seizures, and little to no speech. It is typically caused by a deletion or mutation of the UBE3A gene on chromosome 15, which leads to a lack of functional UBE3A protein in the brain.
A diagnosis of Angelman syndrome may be made based on a detailed patient history, a thorough clinical evaluation, and the identification of characteristic findings. About 80% of cases can be confirmed through a variety of specialized blood tests such as DNA methylation. Fluorescent in situ hybridization (FISH) or, most commonly, microarray chromosome analysis can detect the characteristic deletion of chromosome 15q11-q13 in cells of the body.
Angelman Syndrome Market Key Facts
According to the Angelman Syndrome Foundation (2022), Angelman syndrome is a rare neuro-genetic disorder that occurs in one in 15,000 live births or 500,000 people worldwide. It is caused by a loss of function of the UBE3A gene in the 15th chromosome derived from the mother.
As per Angelman UK (2022), Angelman Syndrome is a rare neurological disorder affecting around 1:20,000 births. Characteristic features include delayed development, severe learning difficulties, little or no speech, and issues with movement and balance.
In a study by the National Institutes of Health Rare Diseases Clinical Research Network Angelman Syndrome Natural History Study (2010), the most common behavioral findings in AS patients were mouthing behavior (95%), short attention span (92%), ataxic or broad-based gait (88%), history of sleep difficulties (80%), and fascination with water (75%).
According to the National Organization for Rare Disorders (2022), Angelman syndrome affects males and females in equal numbers. The prevalence of Angelman syndrome is estimated to be approximately 1 in 12,000-20,000 people in the general population.
The market outlook section of the report helps to build a detailed comprehension of the historical, current, and forecasted market size by analyzing the impact of current and emerging Angelman Syndrome pipeline therapies. It also thoroughly assesses the Angelman Syndrome market drivers & barriers, unmet needs, and emerging technologies set to impact the market dynamics.
The report gives complete details of the market trend for each marketed Angelman Syndrome drug and mid & late-stage pipeline therapies by evaluating their impact based on the annual cost of therapy, their Mechanism of Action (MOA), Route of Administration (ROA), molecule types, competition with other therapies, brand value, and their impact on the market.
Angelman Syndrome Epidemiology Assessment
The epidemiology section provides insights into the historical, current, and forecasted Angelman Syndrome epidemiology trends in the seven major countries (7MM) from 2019 to 2032. It helps to recognize the causes of current and forecasted Angelman Syndrome epidemiology trends by exploring numerous studies and research. The epidemiology section also provides a detailed analysis of diagnosed and prevalent patient pools, future trends, and views of key opinion leaders.
The Report Covers the Angelman Syndrome Epidemiology, Segmented as –
Total Prevalent cases of Angelman Syndrome in the 7MM [2019–2032]
Diagnosed Prevalent Cases of Angelman Syndrome in the 7MM [2019–2032]
Angelman Syndrome Associated with Clinical Manifestations in the 7MM [2019–2032]
Treated Cases of Angelman Syndrome in the 7MM [2019–2032]
Angelman Syndrome Drugs Uptake and Pipeline Development Activities
The drug uptake section focuses on the uptake rate of potential drugs recently launched in the Angelman Syndrome market or expected to be launched during the study period. The analysis covers the Angelman Syndrome market uptake by drugs, patient uptake by therapies, and sales of each drug. Moreover, the therapeutics assessment section helps understand the market dynamics by drug sales, the most rapid drug uptake, and the reasons behind the maximal use of particular drugs. Additionally, it compares the Angelman Syndrome drugs based on their sale and market share.
The report also covers the Angelman Syndrome pipeline development activities. It provides valuable insights about different therapeutic candidates in various stages and the key Angelman Syndrome companies involved in developing targeted therapeutics. It also analyzes recent developments such as collaborations, acquisitions, mergers, licensing patent details, and other information for emerging therapies.
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Angelman Syndrome Therapeutics Analysis
There is no specific therapy for Angelman syndrome at this time. The best treatment is to minimize seizures, anxiety, and gastrointestinal issues and maximize sleep. Thus, the management of Angelman syndrome requires a multidisciplinary approach involving pediatricians, neurologists, geneticists, speech therapists, occupational therapists, physical therapists, and behavioral specialists. Seizures are treated with medications and dietary therapies, while sleep issues are treated with medications and sleep training.
Since the treatment landscape is devoid of any effective curative treatment options, any significant development in this direction is expected to create a tectonic impact on the existing market scenario. Several major pharma and biotech companies are developing therapies for Angelman Syndrome. Some of the drugs in the pipeline include GTX-102 (GeneTx Biotherapeutics/Ultragenyx Pharmaceutical), ION582 (Ionis Pharmaceuticals/Biogen/Emmes Company), and NNZ-2591 (Neuren Pharmaceuticals), among others. Currently, Neuren Pharmaceuticals is leading the therapeutics market with its Angelman Syndrome drug candidates in the most advanced stage of clinical development.
Angelman Syndrome Companies Actively Working in the Therapeutics Market Include
Taysha Gene Therapies
GeneTX Biotherapeutics, LLC
Ultragenyx Pharmaceutical Inc
Ovid Therapeutics Inc.
And Many Others
Emerging and Marketed Angelman Syndrome Therapies Covered in the Report Include:
GT-AS: PTC Therapeutics
GXV 001: GEXVal
RG 6091: Roche
GTX-102: GeneTx Biotherapeutics/Ultragenyx Pharmaceutical
ION582: Ionis Pharmaceuticals/Biogen/Emmes Company
NNZ-2591: Neuren Pharmaceuticals
And Many More
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Table of Content (TOC)
1. Key Insights
2. Executive Summary
3. Angelman Syndrome Competitive Intelligence Analysis
4. Angelman Syndrome Market Overview at a Glance
5. Angelman Syndrome Disease Background and Overview
6. Angelman Syndrome Patient Journey
7. Angelman Syndrome Patient Population and Epidemiology Trends (In the US, EU5, and Japan)
8. Angelman Syndrome Treatment Algorithm, Current Treatment, and Medical Practices
9. Angelman Syndrome Unmet Needs
10. Key Endpoints of Angelman Syndrome Treatment
11. Angelman Syndrome Marketed Therapies
12. Angelman Syndrome Emerging Drugs and Latest Therapeutic Advances
13. Angelman Syndrome Seven Major Market Analysis
14. Attribute Analysis
15. Angelman Syndrome Market Outlook (In US, EU5, and Japan)
16. Angelman Syndrome Companies Active in the Market
17. Angelman Syndrome Access and Reimbursement Overview
18. KOL Views on the Angelman Syndrome Market
19. Angelman Syndrome Market Drivers
20. Angelman Syndrome Market Barriers
22. DelveInsight Capabilities
*The Table of Contents (TOC) is not exhaustive; the final content may vary. Refer to the sample report for the complete table of contents.
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