Galsulfase - Biologic Drug Details

Summary for galsulfase

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

Recent Clinical Trials for galsulfase

Identify potential brand extensions & biosimilar entrants

Sponsor	Phase
BioMarin Pharmaceutical	Phase 4

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Pharmacology for galsulfase

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 galsulfase Derived from Brand-Side Litigation

No patents found based on brand-side litigation

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

No patents found based on company disclosures

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

No patents found based on company disclosures

Market Dynamics and Financial Trajectory for Galsulfase

Introduction to Galsulfase

Galsulfase is a biologic drug used in enzyme replacement therapy (ERT) for the treatment of Mucopolysaccharidosis VI (MPS VI), a rare genetic disorder. It is administered as an intravenous infusion and works by replacing the deficient enzyme, N-acetylgalactosamine 4-sulfatase, which is essential for breaking down certain sugars in the body.

Market Size and Growth

The global enzyme replacement therapy market, which includes galsulfase, is projected to experience significant growth. The market size is expected to increase from $14.61 billion in 2024 to $29.49 billion by 2032, with a Compound Annual Growth Rate (CAGR) of 9.2% during the forecast period[1].

Segmentation and Market Share

In the enzyme replacement therapy market, galsulfase is part of the segment that treats mucopolysaccharidosis (MPS). While the agalsidase segment, which treats Fabry disease, holds a significant market share due to its high cost and increasing demand, galsulfase also contributes substantially to the market due to its specific application in MPS VI[4].

Clinical Benefits and Demand

The demand for galsulfase is driven by its clinical benefits, including improvements in patient mobility and reduction in hospitalizations and surgical procedures. Clinical data show that patients receiving galsulfase can walk farther, climb more stairs, and have fewer hospitalizations compared to those receiving standard medical management (SMM) alone[5].

Cost and Cost-Effectiveness

The cost of galsulfase is a significant factor in its market dynamics. The annual acquisition cost for a patient weighing 25 kg is approximately $399,100, with the total average undiscounted lifetime drug cost per patient estimated to be around $8 million over 20 years[2][5].

Health Economic Evaluation

Health economic evaluations have indicated that while galsulfase provides clinical benefits, its incremental cost-effectiveness ratio is often considered high. For instance, the Australian Pharmaceutical Benefits Advisory Committee concluded that the economic evaluation suggested an unacceptably high incremental cost-effectiveness ratio, although it met the criteria for listing on Australia’s Life Saving Drugs Programme[2][5].

Route of Administration and Market Impact

Galsulfase is administered via the parenteral route, which is expected to grow at the highest CAGR during the forecast period. This route's dominance is due to the majority of ERT drugs, including galsulfase, being available in parenteral formulations[1].

Geographical Market Dynamics

The market for galsulfase is influenced by geographical factors, with North America and Europe being the dominant regions due to favorable reimbursement policies and a strong presence of key market players. Asia Pacific is also expected to grow significantly, driven by increasing awareness and government initiatives[1].

Financial Performance of Key Players

Companies like BioMarin, which markets galsulfase under the brand name Naglazyme, have seen significant financial performance. BioMarin's total revenues have increased, partly due to the growth in sales of their enzyme products, including Naglazyme. However, the financial performance can be affected by factors such as the timing of large government orders and regional market dynamics[3].

Challenges and Opportunities

Despite the high cost, galsulfase presents opportunities for growth due to the increasing prevalence of rare genetic disorders and the lack of alternative treatments. However, challenges include the need for favorable reimbursement policies and the high administrative costs associated with IV infusions[2][5].

Future Outlook

The future outlook for galsulfase is positive, driven by the growing demand for effective treatments for rare diseases. As healthcare infrastructure improves and awareness about enzyme replacement therapies increases, the market for galsulfase is expected to expand. However, addressing the high cost and ensuring cost-effectiveness will remain crucial for its widespread adoption[1][4].

Key Takeaways

• Market Growth: The enzyme replacement therapy market, including galsulfase, is projected to grow significantly, driven by the increasing prevalence of rare genetic disorders.
• High Cost: Galsulfase is associated with a high annual and lifetime cost, which affects its cost-effectiveness.
• Clinical Benefits: The drug offers significant clinical benefits, including improved mobility and reduced hospitalizations.
• Geographical Dynamics: North America and Europe are the dominant regions, with Asia Pacific expected to grow rapidly.
• Financial Performance: Key players like BioMarin have seen revenue growth, but face challenges related to reimbursement and administrative costs.

FAQs

Q: What is galsulfase used for? A: Galsulfase is used in the treatment of Mucopolysaccharidosis VI (MPS VI), a rare genetic disorder.

Q: How is galsulfase administered? A: Galsulfase is administered as an intravenous infusion.

Q: What is the annual cost of galsulfase? A: The annual acquisition cost for a patient weighing 25 kg is approximately $399,100.

Q: What is the estimated lifetime cost of galsulfase? A: The total average undiscounted lifetime drug cost per patient is estimated to be around $8 million over 20 years.

Q: How does galsulfase impact patient health? A: Galsulfase improves patient mobility, reduces hospitalizations, and decreases the need for surgical and diagnostic procedures.

Cited Sources

1. Fortune Business Insights, "Enzyme Replacement Therapy Market Size and Growth [2032]"
2. NCBI, "CADTH CANADIAN DRUG EXPERT COMMITTEE FINAL ... - NCBI"
3. BioMarin, "Earnings Release-31JUL23 EX99.1"
4. Research Nester, "Enzyme Replacement Therapy Market Size, Growth Forecasts 2037"
5. CDA-AMC, "CDR Pharmacoeconomic Review Report for Naglazyme"