Summary

Overview
The global Andersen-tawil syndrome market reached US$ XX million in 2023 and is expected to reach US$ XX million by 2031, growing at a CAGR of XX% 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. Jose 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.
Why Purchase the Report?
- 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.
- Identify commercial opportunities by analyzing trends and co-development
- Excel data sheet with numerous data points of Andersen Tawil syndrome market level with all segments.
- PDF report consists of a comprehensive analysis after exhaustive qualitative interviews and an in-depth study.
- Product mapping is available in excel consisting of key products of all the major players.
The Andersen Tawil Syndrome market report would provide approximately 70 tables, 65 figures, and 186 Pages.
Target Audience 2024
- Manufacturers/ Buyers
- Industry Investors/Investment Bankers
- Research Professionals
- Emerging Companies

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Table of Contents

1. Methodology and Scope
1.1. Research Methodology
1.2. Research Objective and Scope of the Report
2. Definition and Overview
3. Executive Summary
3.1. Snippet by Disease Type
3.2. Snippet by Drug Class
3.3. Snippet by Route of Administration
3.4. Snippet by Distribution Channel
3.5. Snippet by Region
4. Dynamics
4.1. Impacting Factors
4.1.1. Drivers
4.1.1.1. Rise in the Prevalence of Andersen Tawil Syndrome
4.1.1.2. Ongoing Clinical Trials on Andersen Tawil Syndrome
4.1.2. Restraints
4.1.2.1. Complications Associated with Disease
4.1.3. Opportunity
4.1.4. Impact Analysis
5. Industry Analysis
5.1. Porter's Five Force Analysis
5.2. Supply Chain Analysis
5.3. Pricing Analysis
5.4. Regulatory Analysis
5.5. Unmet Needs
5.6. PESTEL Analysis
5.7. Patent Analysis
5.8. SWOT Analysis
6. COVID-19 Analysis
6.1. Analysis of COVID-19
6.1.1. Scenario Before COVID
6.1.2. Scenario During COVID
6.1.3. Scenario Post COVID
6.2. Pricing Dynamics Amid COVID-19
6.3. Demand-Supply Spectrum
6.4. Government Initiatives Related to the Market During the Pandemic
6.5. Manufacturers Strategic Initiatives
6.6. Conclusion
7. By Disease Type
7.1. Introduction
7.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Disease Type
7.1.2. Market Attractiveness Index, By Disease Type
7.2. Type 1*
7.2.1. Introduction
7.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
7.3. Type 2
8. By Drug Class
8.1. Introduction
8.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Drug Class
8.1.2. Market Attractiveness Index, By Drug Class
8.2. Carbonic Anhydrate Inhibitors *
8.2.1. Introduction
8.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
8.3. Antiarrhythmic Drugs
8.4. Amiodarone
8.5. Flecainide
8.6. Beta-Blockers
8.7. Atenolol
9. By Route of Administration
9.1. Introduction
9.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Route of Administration
9.1.2. Market Attractiveness Index, By Route of Administration
9.2. Oral*
9.2.1. Introduction
9.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
9.3. Intravenous
10. By Distribution Channel
10.1. Introduction
10.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Distribution Channel
10.1.2. Market Attractiveness Index, By Distribution Channel
10.2. Hospital Pharmacies*
10.2.1. Introduction
10.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
10.3. Retail Pharmacies
10.4. Online Pharmacies
11. By Region
11.1. Introduction
11.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Region
11.1.2. Market Attractiveness Index, By Region
11.2. North America
11.2.1. Introduction
11.2.2. Key Region-Specific Dynamics
11.2.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Disease Type
11.2.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Drug Class
11.2.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By Route of Administration
11.2.6. Market Size Analysis and Y-o-Y Growth Analysis (%), By Distribution Channel
11.2.7. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
11.2.7.1. U.S.
11.2.7.2. Canada
11.2.7.3. Mexico
11.3. Europe
11.3.1. Introduction
11.3.2. Key Region-Specific Dynamics
11.3.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Disease Type
11.3.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Drug Class
11.3.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By Route of Administration
11.3.6. Market Size Analysis and Y-o-Y Growth Analysis (%), By Distribution Channel
11.3.7. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
11.3.7.1. Germany
11.3.7.2. UK
11.3.7.3. France
11.3.7.4. Italy
11.3.7.5. Spain
11.3.7.6. Rest of Europe
11.4. South America
11.4.1. Introduction
11.4.2. Key Region-Specific Dynamics
11.4.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Disease Type
11.4.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Drug Class
11.4.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By Route of Administration
11.4.6. Market Size Analysis and Y-o-Y Growth Analysis (%), By Distribution Channel
11.4.7. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
11.4.7.1. Brazil
11.4.7.2. Argentina
11.4.7.3. Rest of South America
11.5. Asia-Pacific
11.5.1. Introduction
11.5.2. Key Region-Specific Dynamics
11.5.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Disease Type
11.5.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Drug Class
11.5.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By Route of Administration
11.5.6. Market Size Analysis and Y-o-Y Growth Analysis (%), By Distribution Channel
11.5.7. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
11.5.7.1. China
11.5.7.2. India
11.5.7.3. Japan
11.5.7.4. South Korea
11.5.7.5. Rest of Asia-Pacific
11.6. Middle East and Africa
11.6.1. Introduction
11.6.2. Key Region-Specific Dynamics
11.6.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Disease Type
11.6.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Drug Class
11.6.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By Route of Administration
11.6.6. Market Size Analysis and Y-o-Y Growth Analysis (%), By Distribution Channel
12. Competitive Landscape
12.1. Competitive Scenario
12.2. Market Positioning/Share Analysis
12.3. Mergers and Acquisitions Analysis
13. Company Profiles
13.1. Merck *
13.1.1. Company Overview
13.1.2. Product Portfolio and Description
13.1.3. Financial Overview
13.1.4. Key Developments
13.2. Grevis Pharmaceuticals
13.3. Xeris Pharma
13.4. Novartis AG
13.5. Advanz Pharmaceuticals
13.6. Alembic Pharmaceuticals
13.7. Avet Pharmaceuticals
13.8. Hikma Pharmaceuticals
13.9. Micro Labs
13.10. Advagen Pharma
LIST NOT EXHAUSTIVE
14. Appendix
14.1. About Us and Services
14.2. Contact Us