ASMD is a rare, inherited genetic disorder in which kids are born deficient in an enzyme called acid sphingomyelinase (ASM). This enzyme plays a key role in breaking down a fatty substance called sphingomyelin. A deficiency in this enzyme results in the abnormal accumulation of sphingomyelin in various tissues, especially in cells of the liver, spleen, lungs, brain, and spinal cord. Currently, medications are not available for treating the condition, however bone marrow transplant has shown effectiveness in treating visceral forms of ASMD.

The acid sphingomyelinase deficiency (ASMD) market is estimated to be valued at USD 134.6 Mn in 2024 and is expected to reach USD 300 Mn by 2031, growing at a compound annual growth rate (CAGR) of 12.1% from 2024 to 2031.

Key Takeaways

Key players operating in the ASMD therapeutics market are Genzyme Corporation, Orphazyme A/S, Amicus Therapeutics, and Swedish Orphan Biovitrum AB. Genzyme Corporation’s cerliponase alfa is the only FDA approved treatment for treating neurodegenerative forms of ASMD.

The Acid Sphingomyelinase Deficiency (ASMD) Market is expected to witness high growth over the forecast period owing to increasing prevalence of the disorder and rising research funding for developing novel treatment options. As per estimates, less than 200 people worldwide are known to have ASMD making it an ultra-rare disease.

Technological advancements such as development of new treatment modalities including gene therapies and enzyme replacement therapies (ERTs) are expected to revolutionize the treatment of ASMD in the coming years. Several biotech companies are conducting clinical trials for evaluating efficacy and safety of new treatment options.

Market Trends
Increasing collaboration activities between key market players companies are partnering with research institutes and other pharma giants to jointly develop and market novel treatment therapies.

Growing focus on developing cell and gene therapies with advances in regenerative medicine and gene editing, several companies are exploring gene and cell-based approaches to potentially provide a long-term cure for ASMD through replacing the defective gene or enzyme.

Market Opportunities
Untapped emerging markets in Asia Pacific and Latin America owing to increasing healthcare expenditure and infrastructure expansion in these regions.

Launch of new orphan drugs post positive clinical trial results will open lucrative growth opportunities. Companies developing pipeline candidates are well positioned to tap market potential.

Impact of COVID-19 on ASMD market growth
The COVID-19 pandemic has significantly impacted the growth of the global ASMD market. During the initial lockdown phases in 2020, the market witnessed a decline owing to restrictions on research activities and delayed clinical trials. However, post lockdowns from mid-2021, the market has started recovering steadily as research projects resumed with proper safety protocols. The pandemic highlighted the need for effective diagnosis and treatment of rare genetic disorders like ASMD. Government funding for orphan drug development also increased across major regions. Drug manufacturers focused on digital solutions and homecare support to ensure continuity of care. Teleconsultations helped physicians monitor patients remotely. The long-term impact of COVID-19 is expected to boost market growth through increasing awareness, prioritization of rare disease research, and expansion of telehealth services.

Geographical concentration of ASMD market value
North America dominates the global ASMD market and holds the highest share in terms of value. The region has a well-established healthcare system and growing patient support initiatives that boost early diagnosis and treatment rates. Presence of leading pharmaceutical companies and availability of reimbursement policies also drive the North American market. Europe is the second largest regional market supported by increasing focus on orphan drug development to fulfill unmet needs. Countries like the U.K., Germany, France, and Italy have contributed significantly to the European market growth over the past decade.

Fastest growing region for ASMD market
Asia Pacific region is expected to be the fastest growing market for ASMD over the forecast period. Factors such as rising healthcare expenditure, growing patient volume, and improving access to diagnosis and therapies contribute to the high growth rates. Government efforts to strengthen intellectual property laws to boost innovation also support market expansion. Countries like China, India, Japan, and South Korea offer lucrative opportunities owing to their large patient population and increasing adoption of advanced treatment options. Moreover, ongoing collaborations between regional and international market players will further aid technology transfer and market accessibility across Asia Pacific.

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Ravina Pandya, Content Writer, has a strong foothold in the market research industry. She specializes in writing well-researched articles from different industries, including food and beverages, information and technology, healthcare, chemical and materials, etc.

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