Idursulfase - Biologic Drug Details

Summary for idursulfase

Tradenames:	1
High Confidence Patents:	0
Applicants:	1
BLAs:	1
Suppliers: see list	1
Recent Clinical Trials:	See clinical trials for idursulfase

Recent Clinical Trials for idursulfase

Identify potential brand extensions & biosimilar entrants

Sponsor	Phase
Takeda	Phase 2/Phase 3
Takeda Development Center Americas, Inc.	Phase 4
Takeda	Phase 4

See all idursulfase clinical trials

Pharmacology for idursulfase

Established Pharmacologic Class	Hydrolytic Lysosomal Glycosaminoglycan-specific Enzyme
Chemical Structure	alpha-Glucosidases

Note on Biologic Patents

Matching patents to biologic drugs is far more complicated than for small-molecule drugs.

DrugPatentWatch employs three methods to identify biologic patents:

1. Brand-side disclosures in response to biosimilar applications

These patents were identified from disclosures by the brand-side company, in response to a potential biosimilar seeking to launch. They have a high certainty of blocking biosimilar entry. The expiration dates listed are not estimates — they're expiration dates as indicated by the brand-side company.

2. General brand-side disclosures

These patents were identified from searching drug labels and other general disclosures from the brand-side company. This list may exclude some of the patents which block biosimilar launch, and some of these patents listed may not actually block biosimilar launch. The expiration dates listed for these patents are estimates, based on the grant date of the patent.

3. Patents from broad patent text search

For completeness, these patents were identified by searching the patent literature for mentions of the branded or ingredient name of the drug. Some of these patents protect the original drug, whereas others may protect follow-on inventions or even inventions casually mentioning the drug. The expiration dates listed for these patents are estimates, based on the grant date of the patent.

1) High Certainty: US Patents for idursulfase Derived from Brand-Side Litigation

No patents found based on brand-side litigation

2) High Certainty: US Patents for idursulfase Derived from Company Disclosures

No patents found based on company disclosures

3) Low Certainty: US Patents for idursulfase Derived from Patent Text Search

No patents found based on company disclosures

Market Dynamics and Financial Trajectory for the Biologic Drug: Idursulfase

Introduction

Idursulfase, a biologic drug used in the treatment of Hunter syndrome (Mucopolysaccharidosis II or MPS II), is a crucial enzyme replacement therapy (ERT) that addresses the deficiency of the enzyme iduronate-2-sulfatase. This article delves into the market dynamics and financial trajectory of idursulfase, highlighting its current status, growth prospects, and the factors influencing its market performance.

Hunter Syndrome and the Need for Idursulfase

Hunter syndrome is a rare genetic disorder primarily affecting males, caused by the deficiency of the enzyme iduronate-2-sulfatase. This deficiency leads to the accumulation of glycosaminoglycans (GAGs) in various organs and tissues, resulting in significant physical and cognitive damage over time[1].

Current Market Size and Growth Prospects

The global Hunter syndrome treatment market, led by idursulfase, was estimated at USD 1.31 billion in 2024 and is projected to grow at a CAGR of 5.3% from 2025 to 2030. This growth is driven by increasing awareness of the disease, advancements in therapies, and favorable regulatory environments[1].

Dominance of Enzyme Replacement Therapy (ERT)

Idursulfase, marketed under the brand name Elaprase by Takeda Pharmaceuticals, has been the leading treatment for Hunter syndrome since its approval in 2006. ERT accounts for the largest revenue share, with 57.3% of the market in 2024, due to its efficacy in managing somatic symptoms such as organ enlargement[1].

Clinical Benefits and Outcomes

Studies have shown that intravenous idursulfase treatment results in improved short- and long-term clinical and patient-centered outcomes. These include stabilization or improvement in pulmonary function, reduction in liver and spleen size, and improved urinary GAG levels. Additionally, real-world data have demonstrated increased survival in patients receiving IV idursulfase compared to untreated patients[2][5].

Intrathecal Administration

In addition to IV administration, monthly intrathecal (IT) idursulfase has been evaluated for its potential to prevent cognitive decline and early cognitive impairment in children with MPS II. While the phase 2/3 study did not meet its primary endpoint at 12 months, follow-up data suggest a treatment effect in patients who began idursulfase-IT treatment before the age of 6[2].

Market Drivers

Several factors are driving the growth of the idursulfase market:

Increasing Awareness and Diagnosis

Initiatives such as the #FlyforMPS campaign have increased awareness of Hunter syndrome, leading to better diagnosis and patient management[1].

Research and Development

Rising research and development expenditures are fostering innovation in therapies, particularly in enzyme replacement and gene therapies[1].

Government Funding and Regulatory Support

Government funding for rare disease treatments and favorable regulatory environments are promoting drug development and market growth[1].

Expanding Healthcare Infrastructure

Improving healthcare infrastructure, especially in regions like North America and Europe, is supporting the adoption of idursulfase and other ERTs[4].

Financial Performance and Key Players

The global enzyme replacement therapy market, which includes idursulfase, is projected to grow from $14.61 billion in 2024 to $29.49 billion by 2032 at a CAGR of 9.2%. Major players such as Takeda Pharmaceutical Company Limited, BioMarin, and Sanofi are dominant in this market due to their strong focus on introducing advanced therapy options and expanding their product reach globally[4].

Regional Market Dynamics

• North America: This region holds the largest market share due to the high prevalence of rare lysosomal storage disorders and the strong presence of key market players. The presence of many infusion centers also supports market growth[4].
• Europe: Europe accounts for the second-largest share, driven by favorable reimbursement policies and advancements in healthcare infrastructure[4].
• Asia Pacific: This region is expected to grow significantly due to increasing patient populations and government initiatives to raise awareness about ERT[4].
• Latin America and Middle East & Africa: These regions are expected to grow at a moderate CAGR due to limited infusion centers and reimbursement policies, although there is a growing focus on developing healthcare infrastructure[4].

Challenges and Limitations

Despite the positive market trajectory, there are challenges to consider:

• High Cost: The high cost of idursulfase and other ERTs can be a significant barrier to access, especially in regions with limited reimbursement policies[4].
• Clinical Trial Outcomes: While idursulfase has shown significant clinical efficacy, some clinical trials have faced challenges in meeting regulatory standards, such as the phase 2/3 study for intrathecal idursulfase[2].

Caregiver and Patient Impact

Caregivers and patients have reported improvements in symptoms and quality of life with idursulfase treatment. However, caregivers also face significant strain related to finances and family dynamics, highlighting the need for comprehensive support systems[2].

Key Takeaways

• The global Hunter syndrome treatment market, led by idursulfase, is expected to grow at a CAGR of 5.3% from 2025 to 2030.
• ERT with idursulfase has shown significant clinical efficacy in managing somatic symptoms and improving patient outcomes.
• Increasing awareness, research and development, and favorable regulatory environments are key drivers of market growth.
• Major pharmaceutical companies are expanding their product reach and investing in advanced therapies.
• Regional dynamics vary, with North America and Europe leading the market due to favorable reimbursement policies and healthcare infrastructure.

FAQs

What is the current market size of the Hunter syndrome treatment market?

The global Hunter syndrome treatment market was estimated at USD 1.31 billion in 2024[1].

What is the projected growth rate of the Hunter syndrome treatment market?

The market is projected to grow at a CAGR of 5.3% from 2025 to 2030[1].

What are the primary benefits of idursulfase treatment?

Idursulfase treatment results in improved short- and long-term clinical and patient-centered outcomes, including stabilization or improvement in pulmonary function, reduction in liver and spleen size, and improved urinary GAG levels[2][5].

Which regions are expected to drive the growth of the idursulfase market?

North America and Europe are currently the leading regions, with the Asia Pacific expected to grow significantly in the forecast period[4].

What are the challenges faced by caregivers of patients with Hunter syndrome?

Caregivers experience significant strain linked to finances, family dynamics, and the overall impact of the disease on patients and their families[2].

How does intrathecal idursulfase administration impact cognitive decline in MPS II patients?

Intrathecal idursulfase has shown potential in preventing cognitive decline and early cognitive impairment, particularly in patients who began treatment before the age of 6[2].

Sources

1. Grand View Research: Hunter Syndrome Treatment Market Size, Share Report 2030.
2. PMC: Caregiver experiences and observations of intrathecal idursulfase-IT ...
3. Clinigen Group PLC: ANNUAL REPORT AND ACCOUNTS 2020.
4. Fortune Business Insights: Enzyme Replacement Therapy Market Size and Growth [2032].
5. MDPI: Intravenous Idursulfase for the Treatment of Mucopolysaccharidosis ...

Last updated: 2024-12-17