Acid Sphingomyelinase Deficiency Therapeutics Market is Estimated to Witness High Growth Owing to Promising Drug Pipeline
Acid sphingomyelinase deficiency (ASMD) is a rare genetic disorder characterized by a deficiency of the lysosomal enzyme acid sphingomyelinase, which is responsible for breaking down sphingomyelin. This results in an accumulation of sphingomyelin and other lipids in organs and tissues. Currently, ASMD management involves symptom-specific interventions and therapies. The ASMD therapeutics market is still in nascent stages with no approved drugs. However, a robust drug pipeline with promising candidates in clinical trials is anticipated to drive high growth of this market.
The global Acid Sphingomyelinase Deficiency therapeutics market is estimated to be valued at US$ 30 million in 2024 and is expected to exhibit a CAGR of 25% over the forecast period 2024-2031.
Key Takeaways
Key players operating in the ASMD therapeutics market are Axovant Gene Therapies, Ltd., RegenxBio Inc., and ArmaGen.
The increasing research focusing on developing effective treatments for ASMD presents significant growth opportunities for players in this market. Furthermore, orphan drug designation of candidate therapies is encouraging pharmaceutical companies to invest in the development of ASMD therapeutics.
With a prevalence of 1 in 250,000 live births globally, companies are also eyeing expansion opportunities in major pharmaceutical markets such as the U.S., Europe, and Japan to cater to the needs of affected populations.
Market Drivers
The robust pipeline of ASMD drug candidates in clinical stages is the major factor driving growth of this therapeutics market. Drugs such as AXO-AAV-ASM from Axovant Gene Therapies that has received both FDA orphan drug and rare pediatric disease designations are anticipated to be commercialized in the next 3-5 years, addressing current treatment needs. This promising pipeline is expected to bring effective therapies and provide an impetus to the market.
PEST Analysis
Political: The government policies and regulations related healthcare and clinical research can affect the market growth. Regulations related to approval of therapies will influence the development of treatment options.
Ecnomic: The overall economic growth and spending on healthcare can determine the budget impact on patients, healthcare systems and payers which may positively influence the demand.
Social: Increased awareness about rare diseases and government support groups can help in early diagnosis and better understanding of the condition. This can help patients get access to available treatment options.
Technological: Advancements in understanding the disease pathology at molecular level and development of gene therapy, stem cell therapy and other novel treatment modalities can help address unmet needs and drive the market growth.
Geographical Regions of Concentration
North America region including United States and Canada holds a major share of the global ASMD market in terms of value. This can be attributed to developed healthcare infrastructure, availability of advanced treatment facilities, presence of key market players and supportive patient assistance programs.
Fastest Growing Region
Asia Pacific region is expected to witness the highest growth over the forecast period owing to rising healthcare spending, large patient population, increasing awareness about rare diseases and expanding medical tourism in the region. Improvements in diagnostic facilities and economic growth of countries like China and India are further expected to contribute to the market growth.
*Note:
1. Source: Coherent Market Insights, Public sources, Desk research
2. We have leveraged AI tools to mine information and compile it
