Lysosomal Storage Disease Treatment Market to Reach USD 6.59 Billion by 2032, Driven by Gene Therapy Advancements and Expanded Screening Programs

The global Lysosomal Storage Disease Treatment market size was valued at USD 4.60 billion in 2024 and is projected to reach USD 6.59 billion by 2032, growing at a CAGR of 4.60% during the forecast period of 2025–2032. Market expansion is supported by the long-standing clinical success of enzyme replacement therapies, increasing approvals for rare disease drugs, and growing awareness driven by early diagnosis initiatives.

Advances in biotechnology, coupled with strong regulatory support such as orphan drug and fast-track designations, are accelerating innovation across treatment modalities. The growing focus on one-time, potentially curative gene therapies and improved patient access through home-based and oral treatments continues to enhance long-term market outlook.

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By Therapy Type

Enzyme Replacement Therapy (ERT) dominated the therapy type segment in 2024 and is expected to retain more than 61.3% market share during 2025–2032. ERT remains the cornerstone of treatment for disorders such as Gaucher, Fabry, and Pompe diseases, supported by decades of clinical validation, strong reimbursement frameworks, and widespread physician adoption.

Gene therapy is anticipated to be the fastest-growing segment over the forecast period. The expansion is driven by a robust pipeline of one-time treatments with curative potential, early clinical success in disorders such as MPS II and Krabbe disease, and regulatory incentives including orphan drug and fast-track approvals.

By Application

Gaucher disease led the market in 2024, accounting for 26.7% share, supported by a relatively large treated patient population and multiple approved ERT and SRT options.

Mucopolysaccharidoses (MPS I, II, III, and others) represent the fastest-growing application segment due to rising detection through newborn screening and the urgent need for CNS-targeted therapies. Ongoing clinical trials involving gene transfer and intrathecal enzyme delivery are accelerating therapeutic adoption.

By Route of Administration

Intravenous administration remained dominant with a 68.1% market share in 2024, as most ERTs are delivered via IV infusions in hospital settings.

Oral administration is expanding at the fastest rate, driven by increasing use of oral substrate reduction therapies and chaperone therapies. These formulations improve patient compliance, convenience, and suitability for long-term at-home management.

By End-User

Hospitals accounted for 54.9% of total revenue in 2024, supported by advanced infusion infrastructure, cold-chain capabilities, and specialized monitoring requirements.

Homecare is expected to register the highest CAGR, fueled by patient-centric care models, expanding oral treatment availability, growth in home-based infusion services, and broader insurance coverage integrated with telehealth platforms.

Regional Analysis

North America led the lysosomal storage disease treatment market in 2024, driven by advanced healthcare infrastructure, early adoption of novel therapies, and strong reimbursement systems. The U.S. remains the largest contributor due to FDA-backed accelerated approvals, extensive use of ERTs, and substantial R&D investment. According to NIH data, over USD 2 billion was invested in rare disease research in 2023 alone. Canada is also witnessing increased uptake of SRTs and gene therapies through evolving healthcare policies and patient support programs.

Europe represents the second fastest-growing region, supported by expanded newborn screening, centralized treatment protocols, and strong orphan drug incentives under the EMA. Germany leads the region with high public healthcare spending, comprehensive SHI coverage, and strong clinical trial infrastructure. France and the UK are further strengthening MPS treatment frameworks and expanding oral and home-based therapies. EMA’s PRIME scheme is accelerating gene therapy approvals across the region.

Asia-Pacific is expected to be the fastest-growing market, driven by improving healthcare access, rising disease awareness, and supportive government initiatives for rare disease management. Japan leads with a national rare disease registry, extensive newborn screening, and multiple ERT approvals under the Nanbyo system. China is expanding diagnostics and reimbursement through its NRDL and Healthy China 2030 initiative. Growing adoption of genetic testing and global therapy approvals in India and South Korea is further boosting regional demand.

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Recent Developments

• In 2023–2024, over 35% of late-stage LSD clinical trials globally focused on gene therapy approaches targeting CNS involvement.
• More than 20 orphan drug designations were granted worldwide for lysosomal disorder therapies during the last two years.
• Newborn screening coverage for LSDs expanded by over 15% across developed markets, significantly improving early diagnosis rates.
• Increased adoption of oral therapies contributed to a double-digit rise in homecare-based treatment utilization in key markets.

Key Players

Leading companies in the lysosomal storage disease treatment market include Pfizer Inc., Takeda Pharmaceutical Company Limited, Sanofi (Genzyme), BioMarin Pharmaceutical Inc., Johnson & Johnson (Actelion Pharmaceuticals Ltd), Amicus Therapeutics Inc., Alexion Pharmaceuticals Inc., Ultragenyx Pharmaceutical Inc., JCR Pharmaceuticals Co., Ltd, Orchard Therapeutics, REGENXBIO Inc., Sangamo Therapeutics, AVROBIO Inc., Freeline Therapeutics, Chiesi Farmaceutici S.p.A., Idorsia Pharmaceuticals Ltd, Lysogene S.A., Inventiva Pharma, Orphazyme A/S, and Sigilon Therapeutics Inc.

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