Global Niemann-Pick Disease Type C (NPC) market is expected to reach US$ 923.57 million by 2033 - growing at a CAGR of 28.9%, NPC1 segment holds 92% market revenue

The Global Niemann-Pick Disease Type C (NPC) market reached US$ 9.00 million in 2024 and is expected to reach US$ 923.57 million by 2033, growing at a CAGR of 28.9% during the forecast period of 2025-2033.

The Niemann-Pick Disease Type C market is growing due to rising global awareness, improved genetic screening, and increased diagnosis rates. Advancements in targeted therapies, strong R&D investment, and expanded clinical trials are accelerating innovation. Supportive regulatory pathways and rare disease incentives further drive market expansion and treatment accessibility.

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United States: Key Industry Developments
✅ October 2025: Cyclo Therapeutics completed a merger following shareholder approvals, strengthening its position in NPC treatments, while presenting preliminary Phase III TransportNPC data at events, showing 87% stabilization or improvement in young NPC1 patients on the CGI-C scale at 24 weeks.​

✅ September 2024: MIPLYFFA (arimoclomol) received FDA approval as the first oral treatment for neurological symptoms in NPC patients aged two and older, used alongside miglustat to advance symptomatic management.

Japan Key Industry Developments
✅ July 2025: The European Medicines Agency's CHMP recommended marketing authorization for IntraBio's Aqneursa to treat neurological manifestations of NPC, a decision that could potentially influence future approvals in Japan where only miglustat is currently approved.

✅ July 2025: Azafaros initiated two global Phase III studies for its therapy Nizubaglustat, including NPC patients, which could lead to a new treatment option in Japan if successful

Rising Novel Product Approvals and Development Activities:-
→ Niemann-Pick Disease Type C (NPC) is one of the rarest and most fatal types of lysosomal storage diseases. The incidence of NPC ranges from 1 in 100,000 live births to 1 in 130,000 live births. Up until the 1H of 2024, no specific therapies were approved for NPC, and disease management completely focused on symptomatic relief. However, in the 2H of 2024, two drugs were approved for the indication, which has revolutionized the NPC treatment landscape. These drugs are expected to penetrate various markets around the globe and are anticipated to address the high unmet needs among the diagnosed population.

→ For instance, on September 20, 2024, the U.S. Food and Drugs Administration approved arimoclomol with the brand name MIPLYFFA, manufactured by Zevra Therapeutics, Inc., for the treatment of adult and pediatric NPC patients above 2 years of age. The drug is indicated in combination with Miglustat for the treatment of neurological manifestations of NPC. As per Zevra, nearly 1800 NPC patients were present in the U.S. and Europe, with 300 diagnosed in the United States. Within a month of approval, 90 prescriptions were enrolled in the U.S., and Zevra is extending patient access in Europe through Early Access Programs (EAP) and is planning to reach 70 to 80 patients through this program.

→ On the other hand, within a short span of MIPLYFFA approval, the U.S. FDA also approved IntraBio Inc's AQNEURSA (levacetylleucine) on September 25, 2024, for the treatment of Niemann-Pick Disease Type C (NPC) in the adult and pediatric population. Unlike MIPLYFFA, this drug is approved as monotherapy for the indication. This is another key milestone in the NPC treatment landscape.

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Market Segmenatation-

Segmentation by Disease Type
→ NPC1
This segment includes cases caused by mutations in the NPC1 gene, representing the majority of global NPC diagnoses. NPC1 patients often experience progressive neurological decline, hepatosplenomegaly, and mobility challenges.
It is the dominant disease segment, holding an estimated 92% market share in 2025, driven by higher diagnosis rate, availability of genetic tests, and more active R&D targeting NPC1-specific pathways.

→ NPC2
This segment covers cases linked to NPC2 gene mutations, which are significantly rarer and typically present with rapid onset symptoms in infancy.
NPC2 represents a smaller portion of the market, accounting for approximately 8% share, primarily due to low prevalence and fewer targeted therapeutic programs.

Segmentation by Treatment Type
→ Substrate Reduction Therapy (SRT)
Includes therapies such as miglustat, designed to reduce lipid accumulation in cells. This class remains the primary treatment option in many regions.
SRT holds around 48.5% of the treatment market in 2025 due to established clinical use and access in multiple countries.

→ Supportive/ Symptomatic Care
Supports management of seizures, mobility issues, cognitive decline, and feeding complications. Includes physiotherapy, nutritional support, and neurology care.
This is the second-largest segment, accounting for about 33.2% of the 2025 market, driven by reliance on lifelong supportive interventions.

→ Experimental & Pipeline Therapies
Includes gene therapy, HDAC inhibitors, cyclodextrin-based therapies, and emerging molecular programs. Rapid R&D activity and clinical trials boost this segment's importance.
This category is estimated at 18.3% share in 2025, growing fast as multiple late-stage candidates enter regulatory pathways.

Regional insights:-
→ North America: This region (led by the United States) is the largest regional market for NPC due to advanced rare-disease diagnostics, strong reimbursement frameworks for orphan drugs, high clinical-trial activity, and concentrated presence of key biopharma players and specialty centers. The region benefits from early regulatory pathways (e.g., orphan/drug-approval incentives) that accelerate commercialisation. It is estimated to hold 44.5% of the global NPC market in 2025.

→ Europe: Europe is the second-largest market, driven by established rare-disease networks, pan-European clinical collaborations, growing access programs, and regulatory support across the UK, Germany, France, Italy and other EU countries. Awareness and newborn/paediatric screening pilots in some countries are improving diagnosis rates. Europe's share is estimated at 28.0% in 2025.

→ Asia Pacific (APAC): APAC is a fast-growing region - growth is fuelled by expanding healthcare capacity, rising diagnostic capability, growing clinical trial activity in Japan and China, and increasing investment in rare-disease research. However, diagnosis rates and access to high-cost orphan therapies remain uneven across countries, so APAC's current share is smaller than Western markets but rising. Estimated share in 2025: 16.0%.

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Competitive Landscape:-

The major players in the Niemann-Pick Disease Type C (NPC) market are Zevra Therapeutics, IntraBio, and Johnson & Johnson Services, Inc., among others.

Key Developments
→ On February 7, 2025, Cyclo Therapeutics, Inc. announced the positive preliminary data from its ongoing phase III trial of Trappsol Cyclo, which is designated for the treatment of Niemann-Pick Disease Type C (NPC)1 (NPC1). In 7 among 8 subject populations, Trappsol Cyclo has stabilized and improved the Clinical Global Impression - Severity (CGI-S) scale at 24 weeks, and CGI-S stabilization and improvement was seen in 6 among 7 subjects who received treatment for 48 weeks. The company anticipates interim 48-week data in H1 of 2025, after which regulatory submissions will be done for marketing authorization.

→ On July 16, 2024, Azafaros B.V. announced positive clinical trial results of the phase II trial evaluating nizubaglustat for GM2 gangliosidosis or Niemann-Pick Disease Type C (NPC). The study has shown a positive safety profile of nizubaglustat among the subject population, and upon this success, Azafaros plans to continue pursuing the drug in phase II trials, which are anticipated to begin in 2025.

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