Press Release   -

Barth Syndrome Treatment Market Reach USD 361.56 Billion by 2035

According to analysts at Vantage Market Research, the Global Barth Syndrome Treatment Market size is worth USD 143.34 Billion in 2024 and is projected to reach USD 361.56 Billion by 2035, growing at a CAGR (Compound Annual Growth Rate) of 12.3% from 2024 to 2035. Key trends of market include advancements in enzyme replacement and gene therapies, increased research funding, collaborations with patient advocacy groups, and growing awareness leading to early diagnosis and treatment.

Market Overview 

Barth Syndrome, a rare condition primarily affecting males, is characterized by cardiac issues, skeletal muscle weakness, and neutropenia. As awareness spreads, governments and NGOs are increasingly committed to addressing rare diseases, offering support in the form of research funding, healthcare policies, newborn screening programs, and expanded care facilities. These efforts are helping to identify more cases of Barth Syndrome, thereby increasing the patient population seeking treatment. As a result, the demand for therapies and clinical management options for Barth Syndrome is expected to grow in the coming years.

Given the orphan drug status and the relatively small, profitable market, pharmaceutical companies are often cautious about investing heavily in the development of new treatments independently. However, by partnering with patient advocacy groups, medical technology companies, and other stakeholders, companies can pool their resources and expertise. These partnerships enable more cost-efficient drug discovery, accelerate clinical trials, and allow for sharing of resources, risk, and co-marketing of approved therapies. Collaborating with leading hospitals and clinics worldwide also provides valuable access to specialized clinical data.

U.S. FDA Approves Pfizer’s BEQVEZ™ (fidanacogene elaparvovec-dzkt), a One-Time Gene Therapy for Adults with Hemophilia B

• In April 2024, Pfizer Inc. announced today that the U.S. Food and Drug Administration (FDA) has approved BEQVEZ™ (fidanacogene elaparvovec-dzkt) for the treatment of adults with moderate to severe hemophilia B who currently use factor IX (FIX) prophylaxis therapy, or have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes, and do not have neutralizing antibodies to adeno-associated virus serotype Rh74var (AAVRh74var) capsid as detected by an FDA-approved test

Key Takeaways from the Report

• North America dominated the market with 41.80% market share in 2024. Market is driven by advanced healthcare infrastructure, high awareness of rare diseases, and substantial research and development activities
• Based on the Therapeutic Approaches, the Enzyme replacement therapy category accounted for significant market share of 39.80% in 2024. The segment is aims to provide patients with the deficient tafazzin enzyme using recombinant gene technology
• In 2024, by Drug Classes, Cardiolipin Precursors dominated the market share of 43.20%. The segment is focuses on addressing the root cause of the disorder by replenishing or stabilizing cardiolipin levels
• In 2024, Based on Route of Administration, Oral category dominated the market with market share of 59.50%.  
• By End Users, Hospitals dominated the largest market share. Hospitals play a crucial role as primary healthcare providers for patients requiring specialized care and treatment

Top Companies

• Amneal Pharmaceuticals
• Merck & Co.
• Abbott
• Boehringer Ingelheim
• Medtronic Plc
• Stryker Corporation
• Sanofi
• Pfizer
• F. Hoffman-la Roche
• Mylan Laboratories
• Perrigo Company
• NuVasive
• MicroPort Scientific
• Orthofix
• Johnson and Johnson

Report Coverage

Our market research reports provide comprehensive insights that are essential for strategic decision-making. We cover all key aspects of the market, including dynamics such as drivers, restraints, opportunities, and challenges, alongside the latest industry trends. Our analysis includes an in-depth technology roadmap, product life cycle evaluation, and PESTLE analysis, ensuring a thorough understanding of the market environment. We also assess GDP growth outlooks, examine regional market landscapes, and evaluate the impact of major events like the COVID-19 pandemic. Additionally, our reports feature a detailed competitive landscape, including company market shares and profiles, providing actionable intelligence to empower your business strategies.

Latest News

Stealth Biotherapeutics Announces FDA Acceptance of New Drug Application for Elamipretide for the Treatment of Barth Syndrome

• In April 2024, Stealth BioTherapeutics, a clinical-stage biotechnology company focused on the discovery, development and commercialization of novel therapies for diseases involving mitochondrial dysfunction, today announced that the U.S. Food and Drug Administration (“FDA”) has accepted for filing its New Drug Application (“NDA”) for elamipretide for the treatment of Barth syndrome. The NDA is supported by the positive data from the SPIBA-001 Phase 3 Natural History Control Study and additional supporting efficacy and safety data from the TAZPOWER Part 2 baseline-controlled trial. Elamipretide received Fast Track Designation in 2017, Orphan Drug Designation in 2018 and Rare Pediatric Disease Designation in 2020

About Vantage Market Research:

We, at Vantage Market Research, provide quantified B2B high quality research on more than 20,000 emerging markets, in turn, helping our clients map out constellation of opportunities for their businesses. We, as a competitive intelligence market research and consulting firm provide end to end solutions to our client enterprises to meet their crucial business objectives. Our clientele base spans across 70% of Global Fortune 500 companies. The company provides high quality data and market research services. The company serves various enterprises and clients in a wide variety of industries. The company offers detailed reports on multiple industries including Chemical Materials and Energy, Food and Beverages, Healthcare Technology, etc. The company’s experienced team of Analysts, Researchers, and Consultants use proprietary data sources and numerous statistical tools and techniques to gather and analyse information.