US Angelman Syndrome Therapeutics Market Analysis

US Angelman Syndrome Therapeutics Market Analysis Summary

US Angelman Syndrome Therapeutics Market is valued at around $89.46 Mn in 2022 and is projected to reach $154.87 Mn by 2030, exhibiting a CAGR of 7.1% during the forecast period 2023-2030.

Angelman syndrome is a rare and complicated hereditary disorder that mostly affects the nervous system. Intellectual disability, issues with balance and movement, delayed development, and significant speech impairment are some of this health condition's most prevalent defining symptoms. Recurrent seizures and microcephaly, or small heads, can both occur in a number of affected children. AS is typically diagnosed between the ages of 3 and 7. The Angelman syndrome is expected to afflict more than 1 in 12 to 20 thousand people. There are numerous genetic alterations that cause AS, but they all result in a similar clinical presentation. A loss in the maternally contributed portion of chromosome 15q11–q13 accounts for about 70% of cases. DNA is methylation in the AS crucial region.

Typically, an AS diagnosis and test are performed after the infant is born. The majority of people with AS may be found via a blood test, which determines whether or not the UBE3A gene is operating normally. To identify the syndrome, fluorescent in situ hybridization (FISH) and CGH Array tests are used.

A recent study that disclosed new information about the disease process for Angelman syndrome (AS) found increases in autophagy, a method for removing cellular trash. These findings show that UBE3A deficiency enhances autophagy activity. One of the areas of the brain most affected by AS is the cerebellum. UX111, DTX401, DTX301, and UX701 are just a few of the gene therapy candidates in Ultragenyx's pipeline that have the potential to revolutionize the way many rare genetic illnesses are treated. Recently, the FDA authorized modifications to the GTX-102 Phase 1/2 trial in young patients with Angelman syndrome, enabling Ultragenyx to coordinate dosage scopes and speed up enrolment in the United States.

Boston Children's Hospital, the Angelman Syndrome Foundation, Rady Children's Hospital in San Diego, General Hospital Corporation, Cincinnati Children's Hospital Medical Center, and others are major competitors in the market for treating Angelman syndrome.

Market Dynamics

MarketDrivers

• Rising research and development spending

In the upcoming years, it is projected that the global market for treating Angelman syndrome would grow favourably as research and development spending increases to find better and more efficient treatments.

• Governmental programs

Rising government initiatives from nations across the world in an effort to create new and more effective treatment methods for Angelman syndrome are also projected to support the growth of the global market for treatments for Angelman syndrome in the upcoming years. The Global Angelman Syndrome Registry is an online database run by patient organizations that aim to compile data on the natural history of people with Angelman Syndrome in both children and adults.

• Strengthening Production and Development

Increasing research and development spending has led to the introduction of new technologies and the invention of products in the worldwide market for the treatment of Angelman syndrome.

• Creation of novel and specific

Researchers are currently working to make the treatment process more convenient by using effective treatment and technology.

Market Development

The prevalence of the syndrome has considerably increased around the globe. The first biobank in Australia with the aim of promoting research and treatment of rare genetic diseases caused by changes in genes on chromosome 15—including Prader-Willi Syndrome (PWS) and Angelman Syndrome (AS)—will be created at the Murdoch Children's Research Institute (MCRI).

RARE revealed that the FDA had authorized a protocol amendment after examining the phase I/II research of GTX-102 in young individuals with the uncommon neurogenetic disorder Angelman syndrome. The decision of this regulatory authority will let Ultragenyx to align dose levels in the United States with those being utilized in research cohorts outside of the United States.

"We have 500,000 children estimated to have Angelman syndrome in the globe, and we've identified under 10,000," claims Allyson Berent, chief science officer at the Foundation for Angelman Syndrome Therapeutics (FAST). We've started a number of newborn screening initiatives and a number of global diagnostic efforts for people who are not connected, underdiagnosed, or misdiagnosed in order to help fix that. In spite of the fact that there are currently no approved treatments for the uncommon neurogenetic disorder known as Angelman syndrome, Berent claims that his organization is advancing 13 potential treatments from mice to people, with a number of clinical trials already underway or scheduled to begin in the next 18 to 24 months.

Market Restraints

Children with Angelman syndrome may develop a wide range of neurological symptoms, all of varying degrees of severity. Ataxia, or the inability to coordinate voluntary movements, seizures, trouble sleeping, problems with movement and balance, a limited or impossible ability to converse, and others may also be present. Lack of understanding of this disease not only results in inaccurate diagnosis but also has an effect on the expansion of the global therapeutic market. The market for treatments for Angelman syndrome is also constrained by a paucity of data on the illness's prevalence and financial burden of care.