Acid Sphingomyelinase Deficiency (ASMD), also known as Niemann-Pick disease types A and B, is a rare metabolic disorder characterized by the abnormal accumulation of sphingomyelin in the liver, spleen, lungs and brain due to the reduced or absent activity of the acid sphingomyelinase enzyme. Symptoms of ASMD include enlargement of the liver and spleen, lung disease, and neurological problems. The global ASMD market is driven by the increased research and development of new enzyme replacement therapies which reduces accumulation of sphingomyelin and slows disease progression.
The Global Acid Sphingomyelinase Deficiency therapeutics market is estimated to be valued at US$ 150 million in 2024 and is expected to exhibit a CAGR of 7% over the forecast period 2024-2029. Key Takeaways Key players operating in the Acid Sphingomyelinase Deficiency (ASMD) market are Pfizer Inc, Abeona Therapeutics Inc. and ReGenX Biosciences. Pfizer's enzyme replacement therapy, Cerdelga, is currently the only approved treatment for acid sphingomyelinase deficiency. Several other drugs are in their clinical trial phases. The rising cases of ASMD worldwide and increased investments in R&D of new therapies and drugs are fueling market growth. Technological advancements including development of effective enzyme replacement therapies, targeted therapeutics have improved treatment outcomes in patients with ASMD reducing complications. Market Trends Gene therapy is emerging as a potential treatment for ASMD as it can provide sustained delivery of functional enzymes. Companies are evaluating various gene therapy approaches like hematopoietic stem cell gene therapy. Increased awareness about early diagnosis and treatment through newborn screening programs is helping patients receive optimal care management. Market Opportunities Untapped emerging markets, especially Asia Pacific and Latin America, present major opportunities for existing market players and new entrants. The development of novel drug delivery methods like oral drug formulations can improve patient adherence over intravenous therapies. Combination therapies targeting multiple disease aspects can help achieve better clinical outcomes in ASMD. Impact of COVID-19 on Acid Sphingomyelinase Deficiency (ASMD) market growth The outbreak of COVID-19 pandemic has adversely impacted the growth of global Acid Sphingomyelinase Deficiency (ASMD) market. The pre-COVID situation was positive with rising demand for diagnosis and treatment of ASMD. However, the pandemic led to lockdowns and social distancing norms across various countries. This hampered the routine check-ups, diagnosis procedures and clinical trials related to ASMD. The hospitals shifted their major focus towards handling large number of COVID-19 cases burdening the healthcare infrastructure. This resulted in postponed diagnosis and treatment for other diseases including ASMD. The supply chain disruptions and trade restrictions further affected the production and distribution of diagnostic kits and drugs. With ongoing vaccination drives and relaxation in restrictions in 2021, the ASMD market is recovering slowly. The demand is regaining backed by resumption of diagnostic procedures. The companies are focusing on new product launches, collaborations for clinical trials and capacity expansions. The telehealth and home healthcare are playing a key role in continuing the treatment of chronic diseases amid pandemic. Still uncertainties around new variants poses challenges. Overall, the post-COVID scenario looks positive with market approaching pre-pandemic levels supported by healthcare reforms, funding for research and increasing awareness. Geographical regions where ASMD market value is concentrated: North America represents the largest geographical market for ASMD currently in terms of revenues. The region accounts for over 40% share of global market value due to established healthcare infrastructure, high diagnosis and treatment rates backed by favorable reimbursement system. The key countries contributing heavily include United States and Canada. Europe is also a major region concentrated with around 30% share due to presence of leading pharmaceutical companies and ongoing research activities. Fastest growing region for ASMD market: Asia Pacific region is expected to witness fastest growth for global ASMD market during forecast period. This is owing to rising healthcare expenditure, growing medical tourism industry, increasing voluntary activities by patient advocacy groups and improving economic conditions. In particular, emerging nations like China and India will be the hotspots fueling future market opportunities with their huge population base and developing medical technologies. Though from a smaller base, Asia Pacific region has potential to become second largest ASMD market globally in long run supported by ongoing reforms.
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What Are The Key Data Covered In This Acid Sphingomyelinase Deficiency Therapeutics Market Report?
:- Market CAGR throughout the predicted period
:- Comprehensive information on the aspects that will drive the Acid Sphingomyelinase Deficiency Therapeutics 's growth between 2024 and 2031.
:- Accurate calculation of the size of the Acid Sphingomyelinase Deficiency Therapeutics and its contribution to the market, with emphasis on the parent market
:- Realistic forecasts of future trends and changes in consumer behaviour
:- Acid Sphingomyelinase Deficiency Therapeutics Industry Growth in North America, APAC, Europe, South America, the Middle East, and Africa
:- A complete examination of the market's competitive landscape, as well as extensive information on vendors
:- Detailed examination of the factors that will impede the expansion of Acid Sphingomyelinase Deficiency Therapeutics vendors
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*Note: 1. Source: Coherent Market Insights, Public sources, Desk research 2. We have leveraged AI tools to mine information and compile it
