Agalsidase beta - Biologic Drug Details

Summary for agalsidase beta

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

Recent Clinical Trials for agalsidase beta

Identify potential brand extensions & biosimilar entrants

Sponsor	Phase
ISU Abxis Co., Ltd.	Phase 3
Bio Sidus SA	Phase 3
Genzyme, a Sanofi Company	Phase 3

See all agalsidase beta clinical trials

Pharmacology for agalsidase beta

Established Pharmacologic Class	Hydrolytic Lysosomal Neutral Glycosphingolipid-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 agalsidase beta Derived from Brand-Side Litigation

No patents found based on brand-side litigation

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

These patents were obtained from company disclosures

Applicant	Tradename	Biologic Ingredient	Dosage Form	BLA	Patent No.	Estimated Patent Expiration	Source
Genzyme Corporation	FABRAZYME	agalsidase beta	For Injection	103979	5,356,804	2039-02-26	Company disclosures

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

These patents were obtained by searching patent claims

Applicant	Tradename	Biologic Ingredient	Dosage Form	BLA	Patent No.	Estimated Patent Expiration	Source
Genzyme Corporation	FABRAZYME	agalsidase beta	For Injection	103979	10,016,338	2033-03-11	Patent claims search
Genzyme Corporation	FABRAZYME	agalsidase beta	For Injection	103979	10,111,968	2032-02-15	Patent claims search
Genzyme Corporation	FABRAZYME	agalsidase beta	For Injection	103979	10,182,561	2024-12-29	Patent claims search
Genzyme Corporation	FABRAZYME	agalsidase beta	For Injection	103979	10,280,414	2029-11-17	Patent claims search
Genzyme Corporation	FABRAZYME	agalsidase beta	For Injection	103979	10,543,204	2028-06-26	Patent claims search

International Patents for agalsidase beta

Country	Patent Number	Estimated Expiration
Canada	2094922
Germany	69334327
Portugal	1942189
Spain	2168101
World Intellectual Property Organization (WIPO)	9412628

Supplementary Protection Certificates for agalsidase beta

Supplementary Protection Certificate	SPC Country	SPC Expiration	SPC Description
SZ 20/2010	Austria		PRODUCT NAME: AGALSIDASE ALPHA
2010/021	Ireland		PRODUCT NAME: AGALSIDASE ALFA; NAT REGISTRATION NO/DATE: EU/1/01/188/001-003 20010803; FIRST REGISTRATION NO/DATE: EU/1/01/189/001 03/08/2001 EUROPEAN UNION EU/1/01/188/001-003 03/08/2001 EUROPEAN UNION EU/1/01/189/001 20010803
300452	Netherlands		PRODUCT NAME: AGALSIDASE ALFA; REGISTRATION NO/DATE: EU/1/01/189/001 20010807
300627	Netherlands		PRODUCT NAME: METHOD FOR PRODUCING SECRETED PROTIENS; REGISTRATION NO/DATE: EU/1/01/188/001-006 20010803
1390054-3	Sweden		PRODUCT NAME: AGALSIDAS BETA; REG. NO/DATE: EU/1/01/188/001 20010803

Market Dynamics and Financial Trajectory for Agalsidase Beta

Introduction to Agalsidase Beta

Agalsidase beta, marketed under the brand name Fabrazyme, is a recombinant human alpha-galactosidase A used as an enzyme replacement therapy for the treatment of Fabry disease, a rare genetic disorder characterized by the deficiency of the alpha-galactosidase A enzyme. This enzyme deficiency leads to the accumulation of globotriaosylceramide and other glycosphingolipids, causing severe health complications such as renal failure, cardiomyopathy, and cerebrovascular events[5].

Market Size and Forecast

The global market for Fabrazyme (agalsidase beta) is experiencing significant growth. As of 2023, the market size was estimated at USD 1.2 billion and is projected to reach approximately USD 2.5 billion by 2032, growing at a Compound Annual Growth Rate (CAGR) of 8.2% during the forecast period. This growth is driven by the increasing prevalence of Fabry disease, advancements in biotechnology, and improved diagnostic techniques that have led to higher diagnosis rates[4].

Market Segmentation

The market for agalsidase beta is segmented based on several key factors:

By Application

• The market is segmented into hospital and drug store applications. Hospitals are the primary end-users due to the need for specialized care and infusion facilities for enzyme replacement therapy[1].

By Product

• The product segmentation includes different dosages such as 5mg/vial and 35mg/vial. These dosages are tailored to meet the specific needs of patients based on their condition and treatment plan[1].

By Geography

• The market is geographically segmented into North America, Europe, Asia-Pacific, South America, and the Middle East and Africa. North America and Europe are currently the largest markets due to higher awareness and diagnosis rates of Fabry disease, as well as better healthcare infrastructure[1][4].

Drivers of Market Growth

Several factors are driving the growth of the agalsidase beta market:

Increasing Prevalence of Fabry Disease

• Improved diagnostic techniques and greater awareness among healthcare professionals have led to an increase in the identification of Fabry disease cases, thereby increasing the demand for Fabrazyme[4].

Advancements in Biotechnology

• Continuous advancements in biotechnology have enhanced the production and efficacy of enzyme replacement therapies like agalsidase beta, making them more effective and accessible[4].

Emerging Markets

• The expansion into emerging markets, particularly in Latin America and Asia-Pacific, is expected to contribute significantly to the market growth. For instance, the agreement between mAbxience and Biosidus to manufacture agalsidase beta in Argentina with plans to expand to other international markets highlights this trend[2].

Challenges and Restraints

Despite the positive growth trajectory, the market faces several challenges:

High Cost of Treatment

• The treatment with agalsidase beta is extremely costly, with an average wholesale price of over $300,000 per patient per year. This high cost poses a significant barrier to access, especially in regions with limited healthcare resources[3].

Competition from Biosimilars

• The emergence of biosimilars for agalsidase beta is expected to reduce the market share of the originator product. Biosimilars have the potential to mitigate the effects of future shortages and reduce treatment costs, which could impact the revenue of the original product[3].

Key Players and Collaborations

The market for agalsidase beta involves several key players and strategic collaborations:

Genzyme Corporation

• Genzyme Corporation, the original developer of Fabrazyme, remains a significant player in the market. However, the market is becoming more competitive with the entry of biosimilars and other biopharmaceutical companies[1].

mAbxience and Biosidus

• The recent agreement between mAbxience and Biosidus to manufacture agalsidase beta as a biosimilar is a notable development. This collaboration strengthens mAbxience’s position in the Contract Development and Manufacturing Organization (CDMO) sector and marks a significant milestone for Biosidus in its mission to lead in biotechnology in emerging markets[2].

Financial Impact and Projections

The financial trajectory of the agalsidase beta market is promising, with significant growth projected over the next decade:

Revenue Growth

• The market is expected to grow from USD 1.2 billion in 2023 to approximately USD 2.5 billion by 2032, driven by increasing demand and expanding market reach[4].

Cost-Effectiveness

• Despite the high cost of treatment, the health benefits and improved quality of life for patients with Fabry disease justify the expenditure. However, the introduction of biosimilars is expected to reduce costs and make the treatment more accessible to a broader patient population[3].

Regulatory and Approval Landscape

The regulatory landscape plays a crucial role in the market dynamics of agalsidase beta:

FDA Approval

• Agalsidase beta was granted FDA approval on April 24, 2003, and has since been a critical treatment option for Fabry disease[5].

Biosimilar Approvals

• The approval process for biosimilars is ongoing in various regions, including Japan, where a biosimilar of agalsidase beta is under review. These approvals will likely impact the market share and pricing strategies of the originator product[3].

Conclusion

The market for agalsidase beta is poised for significant growth driven by increasing prevalence of Fabry disease, advancements in biotechnology, and expanding market reach. However, the market also faces challenges such as high treatment costs and competition from biosimilars. Strategic collaborations and the emergence of biosimilars are expected to shape the future of this market.

Key Takeaways

• The global market for Fabrazyme (agalsidase beta) is projected to grow from USD 1.2 billion in 2023 to approximately USD 2.5 billion by 2032.
• The market is driven by the increasing prevalence of Fabry disease and advancements in biotechnology.
• High treatment costs and competition from biosimilars are significant challenges.
• Strategic collaborations, such as the one between mAbxience and Biosidus, are crucial for market expansion.
• Regulatory approvals and the introduction of biosimilars will continue to shape the market dynamics.

FAQs

What is agalsidase beta used for?

Agalsidase beta is used as an enzyme replacement therapy for the treatment of Fabry disease, a rare genetic disorder caused by the deficiency of the alpha-galactosidase A enzyme[5].

How much does the treatment with agalsidase beta cost?

The treatment with agalsidase beta is extremely costly, with an average wholesale price of over $300,000 per patient per year[3].

What are the key drivers of the agalsidase beta market?

The key drivers include the increasing prevalence of Fabry disease, advancements in biotechnology, and expanding market reach into emerging markets[4].

Who are the key players in the agalsidase beta market?

Key players include Genzyme Corporation, mAbxience, and Biosidus. The market is also seeing the entry of biosimilars from various biopharmaceutical companies[1][2].

What is the impact of biosimilars on the agalsidase beta market?

Biosimilars are expected to reduce the market share of the originator product, mitigate future shortages, and reduce treatment costs, making the therapy more accessible to a broader patient population[3].

Sources

1. Global fabrazyme agalsidase beta market size and forecast - Market Research Intellect
2. mAbxience and Biosidus Sign CDMO Agreement for the Manufacture of Agalsidase Beta - mAbxience
3. Biosimilar of High-Cost Agalsidase Beta Under Review in Japan - Center for Biosimilars
4. Fabrazyme (agalsidase beta) Market Report - DataIntelo
5. Agalsidase beta: Uses, Interactions, Mechanism of Action - DrugBank