Acid sphingomyelinase deficiency (ASMD) is a rare lysosomal storage disorder caused by mutations in the sphingomyelin phosphodiesterase 1 (SMPD1) gene. This genetic defect leads to a deficiency in the acid sphingomyelinase enzyme and improper metabolism of sphingomyelin within lysosomes. Patients with ASMD accumulate sphingomyelin in many tissues and organs, which can lead to a wide range of symptoms. Currently, there is no cure for ASMD and management focuses on treating symptoms and complications. However, several pipeline drugs and gene therapies are under research and development which offer hopes of effectively managing the disease.
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 players operating in the acid sphingomyelinase deficiency (ASMD) market are Olipudase alfa (Orphazyme), Taconic Biosciences, Spark Therapeutics.
There is high unmet need for effective therapies for Acid Sphingomyelinase Deficiency (ASMD) Market. Drugs in pipeline offer potential treatment options. Olipudase alfa is under Priority Review by FDA for accelerated approval with PDUFA date of June 2022.
The market is expected to witness significant growth in North America and Europe due to increasing research activities. However, availability of treatment is still limited in other regions.
Market Drivers
Increasing research funding from both private and public organizations is expected to drive the market growth. For instance, National Organization for Rare Disorders (NORD) funds research for rare disease including ASMD. Several ongoing clinical trials and new pipeline candidates in preclinical stage will further boost the market over the forecast period.
PEST Analysis
Political: Government agencies provide funding for research on rare diseases like ASMD. They also consider policy changes that help accelerate drug development and availability.
Economic: Pharmaceutical companies invest heavily in research and clinical trials for developing orphan drugs to treat rare genetic conditions like ASMD. Though the patient pool is small, such drugs fetch high prices and profits.
Social: Patients support groups create awareness and push for more research. They also help patients cope with the condition and access available treatments. Increased patient engagement is seen.
Technological: Researchers are developing gene therapies and other novel treatment approaches. Advances in genetic screening and testing help in early diagnosis. Computer-aided drug discovery aids the research process.
Geographical concentration in terms of value
The major markets where sales of ASMD therapies are concentrated in terms of value currently are North America and Western Europe. Developed countries account for over 80% of total sales value. This is because of higher adoption of expensive orphan drugs in these regions due to greater affordability and accessible healthcare systems.
Fastest growing region
Asia Pacific region is emerging as the fastest growing market for ASMD therapies. This is due to rising healthcare investments, growing population and economic development in major Asian countries. Increased awareness, more screenings and newer treatments getting introduced will fuel growth over the forecast period in Asia Pacific at a CAGR higher than the global average.
Get this Report in Japanese Language: 酸性スフィンゴミエリナーゼ欠損症（ASMD）市場
Get this Report in Korean Language: 산성 스핑고미엘린 분해효소 결핍증(ASMD) 시장
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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. (https://www.linkedin.com/in/ravina-pandya-1a3984191)
