Sanfilippo syndrome, also known as mucopolysaccharidosis type III (MPS III), is a progressive genetic disorder that impairs the body's ability to break down complex sugar compounds called glycosaminoglycans (GAGs). Accumulation of GAGs causes damage to tissues and organs, leading to neurodevelopmental decline and premature death. Currently, there is no cure for Sanfilippo syndrome and available treatments only manage symptoms such as behavioral problems through supportive care. Drugs in clinical trials target the underlying genetic abnormalities to directly slow or stop the accumulation of GAGs in cells. Some of the key players operating in the Sanfilippo syndrome treatment market are Abeona Therapeutics Inc., ArmaGen, Axovant Gene Therapies, Denali Therapeutics, and Regenxbio.
The Global Sanfilippo Syndrome Market is estimated to be valued at USD 9.7 Bn in 2024 and is expected to reach USD 19.8 Bn by 2031, growing at a compound annual growth rate (CAGR) of 9.3% from 2024 to 2031.
Key players operating in the Sanfilippo syndrome treatment market are Abeona Therapeutics Inc., ArmaGen, Axovant Gene Therapies, Denali Therapeutics, and Regenxbio.
Increasing diagnosis rates and growing awareness about Sanfilippo Syndrome Therapeutics Market is driving the demand for treatment. According to a 2018 study, only 30% of patients with Sanfilippo syndrome had received a confirmed diagnosis.
Advancements in gene therapy and development of enzyme replacement therapies are fueling the market growth. Several biotechs are conducting clinical trials evaluating gene therapy candidates delivered by adeno-associated viral vectors to introduce functional copies of deficient enzymes into cells.
Market Trends
Gene therapy is emerging as a promising treatment approach. Axovant Gene Therapies and Regenxbio are conducting separate phase 1/2 clinical trials evaluating intrathecal administration of an AAV9 vector to deliver functional genes.
Partnerships and collaborations are increasing to boost research. In 2021, Abeona Therapeutics partnered with Taysha Gene Therapies to advance AAV-based gene therapies for Sanfilippo syndrome types A, B and B.
Market Opportunities
Obtaining regulatory approvals for gene therapy candidates currently in clinical trials will create commercial opportunities.
Expanding screening efforts to diagnose patients earlier presents an avenue to develop treatments targeting milder forms of the disease.
Impact of COVID-19 on Sanfilippo Syndrome Market Growth
The COVID-19 pandemic has significantly impacted the Sanfilippo syndrome market. During the initial phase of the pandemic, many clinical trials and drug development activities were halted or delayed due to supply chain disruptions and government restrictions. This created uncertainties in the market. However, as the pandemic progressed, companies adopted digital technologies and remote working models to minimize disruptions. Telehealth and home care services emerged as important avenues to support patients.
The spending on research activities also declined initially but is recovering gradually. Many biotech companies are collaborating with academic institutes to accelerate their pipelines and develop treatment alternatives. While the demand for Sanfilippo syndrome drugs witnessed a decline in 2020, it is projected to rise again as economies reopen and awareness increases about managing rare diseases even during crises. The focus is now on ensuring uninterrupted care for patients and supporting new drug discovery via government funding and partnerships.
Geographical Regions with Highest Market Value
North America dominates the global Sanfilippo syndrome market in terms of value, accounting for over 35% market share. This is attributed to the presence of advanced healthcare infrastructure, rising research spending, and growing patient pool in the US and Canada. Moreover, favorable regulations and reimbursement policies drive clinical trials and new product approvals in the region. With increasing diagnosis rates, the North American market is expected to continue its leading position over the forecast period as well.
Fastest Growing Region
Asia Pacific is poised to be the fastest growing regional market for Sanfilippo syndrome globally. This is owing to rapidly developing healthcare sector, growing medical tourism industry, rising patient affordability, and improving access to diagnosis and treatments in major countries like China, India, Japan, and South Korea. Additionally, large population base provides high growth opportunities. Initiatives from governments and international organizations to spread awareness about rare diseases also support market growth in Asia Pacific. The region is estimated to expand at a CAGR of around 11% till 2030.
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