Global andersen-tawil syndrome market - 2024-2031

Global andersen-tawil syndrome market - 2024-2031

The global Andersen-tawil syndrome market reached US$YY million in 2023 and is expected to reach US$YY million by 2031, growing at a CAGR ofYY% during the forecast period 2024-2031.

Andersen-tawil syndrome is an inherited disorder caused by a mutation in the KCNJ2 gene. This gene encodes an ion channel that transports potassium ions out of the cardiac muscle cells. This ion channel is essential for maintaining the normal functions of the skeletal and cardiac muscles. It is usually inherited from a parent in an autosomal dominant manner, but it can also occur due to a new genetic mutation. The specific symptoms and severity can vary greatly from one person to another, even among members of the same family.

Furthermore, the global Anderson-tawil syndrome market is driven by various factors like an increase in the prevalence of Andersen-tawil syndrome, increasing focus of the government to prioritize drugs for the treatment of rare diseases, increasing participation of organizations to support the treatment and to create awareness, rising research and development investments by the key players with having key players like Medtronic Plc, Abbott Laboratories, Boston Scientific Corp, LivaNova Plc and others.

Market Dynamics: Drivers

Ongoing Clinical Trials on Andersen Tawil Syndrome

The ongoing clinical trials on Andersen tawil syndrome help the market to grow during the forecast period, as clinical trials help assess the safety and effectiveness of potential treatments for ATS.

For instance, according to clinicaltrail.gov in September 2022 Centre Hospitalier Universitaire de Liege & University of Liege started a clinical study on Baby Detect Genomic Newborn Screening on which the study objective is to identify 126 treatable severe early onset genetic diseases at birth caused by 361 genes.

These studies are basically for the conditions with, Congenital Adrenal Hyperplasia, Andersen Tawil Syndrome, and others. Hence, this study is estimated to be completed by August 2025.

A research publication in Frontiers in January 2023, states that the ATS with a novel mutation in KCNJ2 is a novel causative variant in KCNJ2 in ATS patients and provided detailed clinical findings to expand the genotype-phenotype correlation. The heterogeneous phenotype of ATS should alert physicians to perform genetic analysis for ATS even in patients with only one or two of the typical triad symptoms.

Restraints

Factors such as complications associated with the disease, ATS has various side effects, including periodic paralysis, which causes muscle weakness or paralysis, primarily in the legs, and can be triggered by exercise, stress, or hot weather. Arrhythmias, abnormal heart rhythms, can cause racing heartbeats, shortness of breath, or fainting, and can be life-threatening. Dysmorphic features, such as low-set ears, wide-spaced eyes, and short stature, are expected to hamper the market.

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Segment Analysis

The Andersen tawil syndrome market is segmented based on disease type, drug class, route of administration, distribution channel, and region.

Segment type 1 accounted for approximately 54.3% of the Andersen Tawil Syndrome market share

The type 1 segment is expected to hold the largest market share over the forecast period. Andersen-Tawil Syndrome type 1 is caused by loss-of-function mutations in the KCNJ2 gene and is associated with facial dysmorphism and a high risk of life-threatening ventricular arrhythmias.

Type 1 Atrial Syndrome (ATS) is caused by mutations in the KCNJ11 gene, affecting 1 in 100,000 to 1 in 1 million people. Symptoms typically appear in childhood or adulthood, with severity varying from person to person.

For instance, in November 2022 Centro Nacional de Investigations Cardiovascular (CNIC) discovered the cause of arrhythmias and sudden death in the rare disease Andersen-Tawil syndrome type 1 (ATS1), which is caused by mutations affecting potassium channels that regulate electrical activity and the intracellular calcium cycle in cardiac and skeletal muscle.

The teams are led by Drs. José Jalife and Juan Antonio Bernal have discovered a previously unknown function of Kir2.1 channels, which control the essential electrical properties of excitable cells such as cardiac muscle, skeletal muscle, and neurons.

In addition, the type 1 atrial syndrome segment is driven by the prevalence of cardiac and dysmorphic features in patients with ATS1. As per a Journal of the American College of Pharmacology research paper in August 2022, Overall, 97% of patients with Andersen-Tawil Syndrome type 1 (ATS1) exhibited cardiac manifestations, (more frequently a large U-wave and ventricular bigeminy), and 75% of patients presented dysmorphic features (more often a small mandible).

As Nature Cardiovascular Research study in October 2022, the ATS1 is manifested as a triad of ventricular arrhythmias, periodic paralysis, and dysmorphic features, and also Kir2.1 dysfunction at the sarcolemma and the sarcoplasmic reticulum causes arrhythmias in a mouse model of Andersen–Tawil syndrome type 1.

Geographical Analysis

North America accounted for approximately 42.5% of the Andersen tawil syndrome market share

North America region is expected to hold the largest market share over the forecast period. Owing to the presence of major key players, advancement in treatment options, well-developed healthcare sector, rising prevalence of disease, and growing awareness towards health in this region. The improvement would be possible because of marketing examination instruments and because of existing players who have positively gotten comfortable with the market since the beginning.

For instance, in 2021, Novartis AG launched a new medication called KEVEYIS (dichlorphenamide) for the treatment of periodic paralysis, a symptom of Andersen-Tawil syndrome.

Furthermore, incentives have been provided by the US government to promote the creation of therapies for uncommon illnesses like ATS. The market for ATS treatments has benefited from these incentives, which have also encouraged more pharmaceutical companies to fund R&D in this field.

Moreover, U.S. government initiatives such as awareness programs would help to drive this market. For instance, in December 2023, the Government introduced the RareCare Patient Assistance Program. It provides patient assistance programs to help individuals living with rare diseases and also provides services such as receiving travel assistance for clinical trials or consultation with disease specialists, connecting with other patient assistance programs, gaining knowledge about rare diseases, and other services.

COVID-19 Impact Analysis

COVID-19 had a substantial impact on the Andersen Tawil Syndrome market and vaccination for patients with neuromuscular disorders evolved during this pandemic in patients having these disorders.

Skeletal muscle channelopathies encompass a group of rare genetic neuromuscular conditions, including myotonia congenita (MC), sodium channel myotonia (SCM), paramyotonia congenita (PMC), hypokalemic periodic paralysis (hypoPP), hyperkalemic periodic paralysis (hyperPP), and Andersen‐Tawil syndrome (ATS). These conditions are characterized by episodic symptoms ranging from myotonia to periodic paralysis caused by the dysfunction of specific ion channels expressed in skeletal muscle.

The impact of coronavirus disease-2019 (COVID-19) infection and vaccination for patients with neuromuscular illnesses is evolving, according to an NCBI publication from October 2024 titled ""COVID-19 infection and vaccination in patients with skeletal muscle channelopathies."" A collection of uncommon genetic neuromuscular disorders known as skeletal muscle channelopathies include Andersen-Tawil syndrome (ATS).

Market Segmentation

By Disease Type
• Type 1
• Type 2

By Drug Class
• Carbonic Anhydrate Inhibitors
Acetazolamide
Dichlorphenamide
• Antiarrhythmic Drugs
• Amiodarone
• Flecainide
• Beta-Blockers
• Atenolol

By Route of Administration
• Oral
• Intravenous
• Others

By Distribution Channel
• Hospital Pharmacies
• Retail Pharmacies
• Online Pharmacies

By Region
• North America
U.S.
Canada
Mexico
• Europe
Germany
U.K.
France
Spain
Italy
Rest of Europe
• South America
Brazil
Argentina
Rest of South America
• Asia-Pacific
China
India
Japan
South Korea
Rest of Asia-Pacific
• Middle East and Africa

Competitive Landscape

The major global players in the Andersen tawil syndrome market include Merck, Grevis Pharmaceuticals, Xeris Pharma, Novartis AG, Advanz Pharmaceuticals, Alembic Pharmaceuticals, Avet Pharmaceuticals, Hikma Pharmaceuticals, Micro Labs, Advagen Pharma, and among others among others.

Key Developments

 On May 6, 2024, Xeris Biopharma Holdings, Inc. announced that it has entered into an exclusive worldwide collaboration and license agreement with Beta Bionics, Inc. for the development and commercialization of a glucagon product utilizing Xeris, XeriSol technology for use in Beta Bionics’ proprietary bi-hormonal pump and pump systems.

 On May 6, 2024, Xeris Biopharma Holdings, Inc. announced that their product KEVEYIS is the first FDA-approved treatment for Primary Periodic Paralysis.

 On May 12, 2022, a French study, involving clinicians from the Institute, was carried out on 35 patients with ATS whose diagnosis was confirmed by molecular analysis. They were diagnosed and are being followed in Neuromuscular reference centers in France. The study included a retrospective clinical, histological, electroneuromyography (ENMG), and genetic analysis.

 On April 1, 2021, Teva Pharmaceuticals was acquired by Karo Pharma Aktiebolag to gain the European OTC brand portfolios and drug research including rare disease solutions. Also, Sun Pharmaceutical Industries Ltd. announced the acquisition of a 66% ownership interest in Trikaal Medi infotech, and AWACS, through its subsidiary ABCD Technologies LLP.

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• To visualize the Andersen Tawil syndrome market segmentation based on disease type, drug class, route of administration, distribution channel and region as well as understand key commercial assets and players.
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Target Audience 2024
• Manufacturers/ Buyers
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• Research Professionals
• Emerging Companies