February 16, 2025
Acid Sphingomyelinase Deficiency Therapeutics Market

Acid Sphingomyelinase Deficiency Therapeutics Market is driven by Gene Therapy Trends

The acid sphingomyelinase deficiency (ASMD) is a rare genetic lysosomal storage disorder caused due to the deficiency of the acid sphingomyelinase enzyme. This deficiency leads to excessive accumulation of sphingomyelin, especially in tissues like the spleen, liver and lungs. The prominent symptoms of ASMD include enlarged spleen and liver, lung, skeletal and immune system complications. Currently, there is no definitive cure for ASMD and treatment involves managing symptoms. However, gene therapy is emerging as a potential treatment option that aims to replace the defective gene.

The global Acid Sphingomyelinase Deficiency therapeutics market is estimated to be valued at US$ 50 million in 2030 and is expected to exhibit a CAGR of 7% over the forecast period 2028-2030.

Key Takeaways

Key players operating in the Acid Sphingomyelinase Deficiency therapeutics market are clinical-stage biotech companies like ArmaGen, REGENXBIO and Sangamo Therapeutics. ArmaGen is developing AGLE-177, an AAV-based gene therapy for ASMD, currently in Phase I/II clinical trials. REGENXBIO’s RGX-111, a potential one-time gene therapy for ASMD, received FDA orphan drug designation in 2020.
The market is witnessing growing demand driven by rising ASMD patient diagnoses supported by newborn screening programs. Additionally, the approval of the first disease-modifying therapies is expected to significantly improve treatment outcomes.

Manufacturers are also expanding globally with strategic partnerships and collaborations. For instance, Sangamo inked a worldwide deal with Pfizer in 2019 to develop and commercialize ST-400 gene therapy for ASMD outside the U.S. and Canada. Such collaborations will boost global access to innovative ASMD treatments.

Market key trends
Emergence of gene therapy as a promising treatment approach is a major trend in the ASMD market. Unlike conventional therapies that only manage symptoms, gene therapies aim to correct the underlying genetic defect. As the first gene therapy RGX-111 entered Phase I/II trials, it represents a potential one-time curative option if proven effective and safe. Further research is ongoing to optimize gene therapy vectors, dosing and delivery to maximize therapeutic benefits. This represents tremendous hope for ASMD patients with no existing cure.

Porter’s Analysis

Threat of new entrants: Rare genetic condition has limited market potential due to small patient population.

Bargaining power of buyers: High unmet medical need provides leverage for patients and advocacy groups in negotiating treatment price with manufacturers.

Bargaining power of suppliers: Limited competition between drug developers currently targeting ASMD provides suppliers greater influence over buyers.

Threat of new substitutes: No substitute therapies currently exist for ASMD treatment, providing existing treatments with insulation from substitute threats.

Competitive rivalry: With a sole approved therapy and clinical-stage competitors, competition is currently constrained but expected to intensify as more treatments enter the market. The United States and Western Europe represent the largest geographical markets for ASMD treatments in terms of value currently. This is due to the concentration of diagnosed patients as well as greater economic ability to pay for high-cost orphan drugs in these developed regions.

China and other countries in Asia Pacific are poised to become the fastest growing regional markets over the long term. Heightened diagnosis rates and rising incomes are expected to drive increased healthcare spending and uptake of ASMD therapies in emerging Asian countries in the coming years. Additionally, multinational pharmaceutical companies are anticipated to proactively register orphan drugs in these high-potential growth regions.

*Note
1. Source: Coherent Market Insights, Public sources, Desk research
2. We have leveraged AI tools to mine information and compile it

Ravina
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Ravina Pandya, a 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, chemicals and materials, etc. With an MBA in E-commerce, she has expertise in SEO-optimized content that resonates with industry professionals. 

Ravina Pandya

Ravina Pandya, a 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, chemicals and materials, etc. With an MBA in E-commerce, she has expertise in SEO-optimized content that resonates with industry professionals. 

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