Angelman Syndrome Market to Witness Significant Growth Through 2034, Driven by Novel Therapeutic Advancements | DelveInsight

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Jun 02, 2025, 17:31 ET

According to DelveInsight's estimates, the Angelman syndrome market size in the 7MM is expected to show positive growth during the forecast period (2025–2034), primarily driven by the increasing prevalence of Angelman syndrome, growing awareness and diagnosis, and new product launches.

LAS VEGAS, June 2, 2025 /PRNewswire/ -- Angelman syndrome is a rare neurogenetic disorder affecting approximately 1 in 15,000 live births or 500,000 people globally. It results from the loss of function of the UBE3A gene located on the 15th chromosome, specifically the copy inherited from the mother. This loss is typically caused by a deletion or mutation of the UBE3A gene, leading to a deficiency of functional UBE3A protein in the brain.

The condition is typically identified by developmental delays that become apparent between 6 and 12 months of age. Both males and females are equally affected, with the Angelman syndrome prevalence estimated to be about 1 in 12,000 to 20,000 people in the general population.

In the US, the Angelman syndrome epidemiology is characterized by a diverse landscape. While rare, the Angelman syndrome prevalence is expected to rise due to improvements in diagnostic techniques, expanded availability of genetic testing, and greater awareness among healthcare professionals and the general public. This condition presents with developmental delays, intellectual disabilities, significant speech impairments, and distinct behavioral traits.

As Angelman syndrome treatment options improve, along with advancements in supportive services and increased life expectancy for those affected, the number of individuals diagnosed is anticipated to grow. This evolving landscape highlights the importance of continued research, early detection, and comprehensive care to better support individuals with Angelman syndrome and their families.

Learn more about the Angelman syndrome epidemiology @ Angelman Syndrome Cases

Current treatment approaches for Angelman syndrome focus on a comprehensive, multidisciplinary strategy to manage symptoms and improve quality of life. Although there is no cure, treatments like antiepileptic drugs for seizure control and behavioral therapies are key in symptom management. Augmentative and alternative communication methods, alongside physical and occupational therapy, address developmental delays and enhance functional skills. Support services, including special education and support groups, provide vital assistance for individuals and families affected by Angelman syndrome. Current research on Angelman syndrome into experimental therapies, such as gene therapy and targeted drug treatments, offers promise for future breakthroughs in managing the condition.

Angelman syndrome medications like RISPERDAL (risperidone) may help control hyperactive and socially disruptive behaviors, but should not be used long-term. Acid reflux (gastroesophageal reflux) is common in individuals with Angelman syndrome and can be managed by keeping the person upright during and after meals or using Angelman syndrome medications like Gaviscon and ranitidine. In severe, rare cases, a surgical procedure called fundoplication may be necessary.

While current Angelman syndrome treatments focus on managing symptoms, there is a significant need for disease-modifying therapies that address the underlying genetic cause of Angelman syndrome, such as gene therapy or UBE3A reactivation strategies.

Find out more about Angelman syndrome therapy @ Angelman Syndrome Medication

Despite available antiepileptic Angelman syndrome medications, many individuals with Angelman syndrome experience treatment-resistant seizures, highlighting the need for more effective seizure management options. While augmentative and alternative communication (AAC) methods are helpful, there is a need for more advanced and accessible communication aids to improve communication abilities in non-verbal individuals.

There is a need for long-term support services to address the evolving needs of individuals with Angelman syndrome as they transition into adulthood, including vocational training, independent living support, and access to specialized healthcare services.

The pipeline of emerging treatments for Angelman syndrome shows promise, with mid-late stage products such as Ionis Pharmaceuticals' ION582, Ultragenyx Pharmaceuticals' GTX-102, Neuren Pharmaceuticals' NNZ-2591, MavriX Bio's MVX-220, and others demonstrating the potential to significantly impact the Angelman syndrome market and potentially revolutionize the treatment approach.

Discover which Angelman syndrome therapy will grab major market share @ Angelman Syndrome Medical Research

ION582 is an experimental antisense drug aimed at inhibiting the UBE3A transcript (UBE3A-ATS) and enhancing the production of UBE3A protein, potentially offering a treatment for Angelman syndrome. In July 2024, Ionis Pharmaceuticals reported positive outcomes from the HALOS Phase I/II study of ION582 in Angelman syndrome. Notable improvements were observed in communication, cognition, and motor function, with 97% of participants in the medium and high-dose groups showing overall symptom improvement based on SAS-CGI-C assessments. Ionis plans to start Phase III development in the first half of 2025.

In May 2024, Ionis revealed positive topline results from the HALOS Phase I/IIa open-label study of ION582 in Angelman syndrome and announced that it would independently advance ION582 as part of its portfolio of potentially transformative treatments for serious neurological conditions. The company also plans to consult with regulatory authorities on the design of a pivotal program. Biogen chose not to exercise its option to license ION582. In 2022, the US FDA and EMA granted Orphan Drug Designation to ION582, and the FDA also awarded Fast Track and Rare Pediatric Designation. The drug is currently in Phase II trials and progressing toward Phase III.

GTX-102 is an experimental antisense oligonucleotide delivered via intrathecal administration, targeting and inhibiting the expression of UBE3A-AS. Preclinical studies suggest that GTX-102 reduces UBE3A-AS levels and reactivates the paternal UBE3A allele in CNS neurons. GTX-102 has received Orphan Drug Designation, Rare Pediatric Disease Designation, and Fast Track Designation from the FDA, as well as Orphan Designation and PRIME designation from the EMA.

In July 2024, Ultragenyx Pharmaceuticals presented new data and regulatory updates on GTX-102 at the ASF Conference, reviewing Phase I/II results and outlining plans to begin Phase III trials by the end of the year. In August 2019, GeneTx and Ultragenyx formed a partnership to develop GTX-102, with Ultragenyx acquiring exclusive rights to GeneTx in July 2022 for an initial payment of USD 75 million, plus future milestone and royalty payments. In April 2024, Ultragenyx announced positive interim Phase I/II data in Angelman syndrome patients treated with GTX-102. The drug is currently in Phase III trials.

NNZ-2591 is an enhanced synthetic version of a naturally occurring brain peptide related to IGF-1, a growth factor stimulated by growth hormone. The drug is being developed to treat Angelman syndrome by restoring the natural balance of brain function. Neuren has received Orphan Drug Designation for NNZ-2591 from both the US FDA and EMA. The company has recently completed a Phase II clinical trial for Angelman syndrome. In August 2024, Neuren released positive top-line results from its Phase II trial, showing that the drug was safe and well-tolerated, with no serious adverse events. Furthermore, significant improvements were observed in both clinician and caregiver global efficacy measures tailored to the disease.

MVX-220 is an experimental gene therapy based on the hu68AAV vector, developed to deliver the human UBE3A gene directly to brain neurons through a single intra-cisterna magna (ICM) injection. In preclinical studies, it has demonstrated the ability to restore UBE3A protein levels and alleviate symptoms in mouse models of Angelman syndrome. The therapy will be assessed in the Phase I/II clinical trial known as ASCEND-AS (AAV-mediated expression in neurons for Angelman syndrome).

In May 2025, MavriX Bio announced that the FDA approved its Investigational New Drug (IND) application for MVX-220. The company plans to begin the first-in-human (FIH) ASCEND-AS clinical trial in the second half of 2025.

To know about what is the current status of research on Angelman syndrome, visit @ Angelman Syndrome Cure Research

The anticipated launch of these emerging therapies for Angelman syndrome are poised to transform the market landscape in the coming years. As these cutting-edge therapies continue to mature and gain regulatory approval, they are expected to reshape the Angelman syndrome market landscape, offering new standards of care and unlocking opportunities for medical innovation and economic growth.

DelveInsight estimates that the market size for Angelman syndrome is expected to grow at a significant CAGR by 2034. This growth is mainly attributed to the increasing prevalence of Angelman syndrome, growing awareness and diagnoses, and the new product launches.

DelveInsight's latest published market report, titled as Angelman Syndrome Market Insight, Epidemiology, and Market Forecast – 2034, will help you to discover which market leader is going to capture the largest market share. The report provides comprehensive insights into the Angelman syndrome country-specific treatment guidelines, patient pool analysis, and epidemiology forecast to help understand the key opportunities and assess the market's underlying potential. The Angelman syndrome market report proffers epidemiological analysis for the study period 2020–2034 in the 7MM segmented into:

Total Prevalence of Angelman Syndrome
Total Diagnosed Prevalence of Angelman Syndrome
Angelman Syndrome Cases Associated with Clinical Manifestations
Treated Cases of Angelman Syndrome

The report provides an edge while developing business strategies by understanding trends shaping and driving the 7MM Angelman syndrome market. Highlights include:

10-year Forecast
7MM Analysis
Epidemiology-based Market Forecasting
Historical and Forecasted Market Analysis upto 2034
Emerging Drug Market Uptake
Peak Sales Analysis
Key Cross Competition Analysis
Industry Expert's Opinion
Access and Reimbursement

Download this Angelman syndrome market report to assess the epidemiology forecasts, understand the patient journeys, know KOLs' opinions about the upcoming treatment paradigms, and determine the factors contributing to the shift in the Angelman syndrome market. Also, stay abreast of the mitigating factors to improve your market position in the Angelman syndrome therapeutic space.

Angelman Syndrome Epidemiology Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted Angelman syndrome epidemiology in the 7MM, i.e., the United States, EU5 (Germany, Spain, Italy, France, and the United Kingdom), and Japan.

Angelman Syndrome Pipeline

Angelman Syndrome Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key Angelman syndrome companies, including Neuren Pharmaceuticals, Ultragenyx Pharmaceutical, Ionis Pharmaceuticals, Roche, GEXVal, PTC Therapeutics, Taysha Gene Therapies, Ovid Therapeutics, among others.

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Rett Syndrome Pipeline

Rett Syndrome Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key Rett syndrome companies, including Anavex Life Sciences Corp, Neurogene, Taysha GTx, among others.

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SOURCE DelveInsight Business Research, LLP