X-Linked Myotubular Myopathy Market

Market Overview:

The 7 major x-linked myotubular myopathy markets reached a value of USD 2,177.1 Million in 2024. Looking forward, IMARC Group expects the 7MM to reach USD 3,423.3 Million by 2035, exhibiting a growth rate (CAGR) of 4.23% during 2025-2035.

The x-linked myotubular myopathy market has been comprehensively analyzed in IMARC's new report titled "X-Linked Myotubular Myopathy Market: Epidemiology, Industry Trends, Share, Size, Growth, Opportunity, and Forecast 2025-2035". X-linked myotubular myopathy (XLMTM) is an autosomal recessive, severe neuromuscular disease due to mutations in the MTM1 gene encoding myotubularin, a protein required for healthy muscle fiber growth and maintenance. Lack or dysfunction of myotubularin results in abnormal muscle development, which causes extreme weakness of the muscles and diminished motor function. XLMTM is found predominantly in male infants because the MTM1 gene resides on the X chromosome and phenotypes usually appear at birth or early in infancy. Severe hypotonia, ventilatory support for respiratory insufficiency, feeding intolerance, and delayed motor milestones characterize affected individuals. Facies and limb weakness are also present, leading to restricted mobility. Diagnosis is by clinical examination, genetic sequencing, electromyography (EMG), and muscle biopsy to establish MTM1 mutations and muscle pathology. No cure is available, but supportive care including respiratory care, physical therapy, and new gene therapies are under investigation to enhance outcomes and survival.

The growing interest in gene therapy as a potential cure is a prime driver for the X-linked myotubular myopathy (XLMTM) market. Gene therapy, especially experimental products such as resamirigene bilparvovec (previously AT132), seeks to reinstate MTM1 gene activity, thus halting the primary cause of the disease. Initial clinical results have shown encouraging enhancement in muscle power and respiratory support, bringing the promise of revolutionary treatment. In addition, the increasing frequency of neonatal respiratory distress, a major complication of XLMTM, is stimulating research into improved supportive care, including sophisticated ventilatory support and neuromuscular rehabilitation. Increased newborn screening programs and genetic testing are also stimulating early diagnosis, thus allowing early intervention. More importantly, increased joint efforts between pharmaceutical firms and patient advocacy groups are propelling research, increasing awareness, and enhancing access to clinical trials. With regulatory bodies making rare disease drugs a top priority, faster approvals and supportive payment policies are likely to improve accessibility of treatment, complementing market expansion.

IMARC Group's new report provides an exhaustive analysis of the x-linked myotubular myopathy market in the United States, EU4 (Germany, Spain, Italy, and France), United Kingdom, and Japan. This includes treatment practices, in-market, and pipeline drugs, share of individual therapies, market performance across the seven major markets, market performance of key companies and their drugs, etc. The report also provides the current and future patient pool across the seven major markets. Furthermore, the current treatment practice/algorithm, market drivers, challenges, opportunities, reimbursement scenario, unmet medical needs, etc., have also been provided in the report. This report is a must-read for manufacturers, investors, business strategists, researchers, consultants, and all those who have any kind of stake or are planning to foray into the x-linked myotubular myopathy market in any manner.

Recent Developments:

• In October 2024, a European study reported a three-year follow-up of 26 patients, showing a general decline in muscle strength and motor function over time, with minimal improvements in ambulatory abilities. These findings underscore the progressive nature of XLMTM and highlight the critical need for effective therapeutic interventions.
• In November 2023, The Lancet Neurology published results from the ASPIRO trial, revealing that 16 of 24 children with X-linked myotubular myopathy achieved independent breathing post-gene therapy.

Key Highlights:

• XLMTM affects approximately 1 in 50,000 male live births worldwide.
• The overall mortality rate is 47%, with respiratory failure being the leading cause of death.
• Approximately 87% of affected males require respiratory support at birth.
• Around 86.7% of individuals with XLMTM are non-ambulant, relying on wheelchairs for mobility.
• An unexpectedly high incidence of learning disabilities is observed in 43% of patients.

Drugs:

AT132 is an investigational gene therapy for X-linked myotubular myopathy (XLMTM), delivering a functional MTM1 gene via an AAV8 vector to improve muscle function. Clinical studies have shown potential benefits, including reduced ventilator dependence and achievement of motor milestones. However, serious adverse events, including liver complications, have been reported.

Time Period of the Study

• Base Year: 2024
• Historical Period: 2019-2024
• Market Forecast: 2025-2035

Countries Covered

• United States
• Germany
• France
• United Kingdom
• Italy
• Spain
• Japan

Analysis Covered Across Each Country

• Historical, current, and future epidemiology scenario
• Historical, current, and future performance of the x-linked myotubular myopathy market
• Historical, current, and future performance of various therapeutic categories in the market
• Sales of various drugs across the x-linked myotubular myopathy market
• Reimbursement scenario in the market
• In-market and pipeline drugs

Competitive Landscape:

This report also provides a detailed analysis of the current x-linked myotubular myopathy marketed drugs and late-stage pipeline drugs.

In-Market Drugs

• Drug Overview
• Mechanism of Action
• Regulatory Status
• Clinical Trial Results
• Drug Uptake and Market Performance

Late-Stage Pipeline Drugs

• Drug Overview
• Mechanism of Action
• Regulatory Status
• Clinical Trial Results
• Drug Uptake and Market Performance
   

*Kindly note that the drugs in the above table only represent a partial list of marketed/pipeline drugs, and the complete list has been provided in the report.

Key Questions Answered in this Report:

Market Insights

• How has the x-linked myotubular myopathy market performed so far and how will it perform in the coming years?
• What are the markets shares of various therapeutic segments in 2024 and how are they expected to perform till 2035?
• What was the country-wise size of the x-linked myotubular myopathy market across the seven major markets in 2024 and what will it look like in 2035?
• What is the growth rate of the x-linked myotubular myopathy market across the seven major markets and what will be the expected growth over the next ten years?
• What are the key unmet needs in the market?

Epidemiology Insights

• What is the number of prevalent cases (2019-2035​) of x-linked myotubular myopathy across the seven major markets?
• What is the number of prevalent cases (2019-2035​) of x-linked myotubular myopathy by age across the seven major markets?
• What is the number of prevalent cases (​2019-2035​) of x-linked myotubular myopathy by gender across the seven major markets?
• How many patients are diagnosed (​2019-2035​) with x-linked myotubular myopathy across the seven major markets?
• What is the size of the x-linked myotubular myopathy patient pool (2019-2024) across the seven major markets?
• What would be the forecasted patient pool (2025-2035) across the seven major markets?
• What are the key factors driving the epidemiological trend of x-linked myotubular myopathy?
• What will be the growth rate of patients across the seven major markets?

X-Linked Myotubular Myopathy: Current Treatment Scenario, Marketed Drugs and Emerging Therapies

• What are the current marketed drugs and what are their market performance?
• What are the key pipeline drugs and how are they expected to perform in the coming years?
• How safe are the current marketed drugs and what are their efficacies?
• How safe are the late-stage pipeline drugs and what are their efficacies?
• What are the current treatment guidelines for x-linked myotubular myopathy drugs across the seven major markets?
• Who are the key companies in the market and what are their market shares?
• What are the key mergers and acquisitions, licensing activities, collaborations, etc. related to the x-linked myotubular myopathy market?
• What are the key regulatory events related to the x-linked myotubular myopathy market?
• What is the structure of clinical trial landscape by status related to the x-linked myotubular myopathy market?
• What is the structure of clinical trial landscape by phase related to the x-linked myotubular myopathy market?
• What is the structure of clinical trial landscape by route of administration related to the x-linked myotubular myopathy market?