ALDURAZYME Drug Profile

Summary for Tradename: ALDURAZYME

High Confidence Patents:	0
Applicants:	1
BLAs:	1
Recent Clinical Trials:	See clinical trials for ALDURAZYME

Recent Clinical Trials for ALDURAZYME

Identify potential brand extensions & biosimilar entrants

Sponsor	Phase
UCSF Benioff Children's Hospital Oakland	N/A
Children's Hospital & Research Center Oakland	N/A
agnes chen	N/A

See all ALDURAZYME clinical trials

Pharmacology for ALDURAZYME

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 ALDURAZYME Derived from Brand-Side Litigation

No patents found based on brand-side litigation

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

No patents found based on company disclosures

3) Low Certainty: US Patents for ALDURAZYME 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
Biomarin Pharmaceutical Inc.	ALDURAZYME	laronidase	Injection	125058	10,016,338	2033-03-11	Patent claims search
Biomarin Pharmaceutical Inc.	ALDURAZYME	laronidase	Injection	125058	10,111,968	2032-02-15	Patent claims search
Biomarin Pharmaceutical Inc.	ALDURAZYME	laronidase	Injection	125058	10,182,561	2024-12-29	Patent claims search
Biomarin Pharmaceutical Inc.	ALDURAZYME	laronidase	Injection	125058	10,316,282	2033-03-19	Patent claims search
Biomarin Pharmaceutical Inc.	ALDURAZYME	laronidase	Injection	125058	10,577,154	2029-05-13	Patent claims search
Biomarin Pharmaceutical Inc.	ALDURAZYME	laronidase	Injection	125058	10,668,053	2033-05-03	Patent claims search
Biomarin Pharmaceutical Inc.	ALDURAZYME	laronidase	Injection	125058	10,717,958	2033-03-19	Patent claims search

ALDURAZYME Market Analysis and Financial Projection Experimental

Market Dynamics and Financial Trajectory for the Biologic Drug: ALDURAZYME

Introduction

ALDURAZYME, a recombinant human alpha-L-iduronidase enzyme, is a biologic drug developed by BioMarin Pharmaceutical Inc. for the treatment of mucopolysaccharidosis I (MPS I), a rare genetic disorder. Here, we will delve into the market dynamics and financial trajectory of ALDURAZYME, highlighting key performance indicators, market trends, and future outlook.

Historical Performance

Early Years and Approval

ALDURAZYME was approved by the FDA in 2003, marking a significant milestone in the treatment of MPS I. Since its approval, the drug has been a cornerstone in the management of this rare disease.

Revenue Growth

In the early years following its approval, ALDURAZYME showed steady revenue growth. For instance, in 2010, ALDURAZYME net product revenue was $71.2 million, which was part of BioMarin's total net product revenue of $369.7 million[2].

Quarterly and Annual Performance

In recent years, ALDURAZYME has continued to contribute significantly to BioMarin's revenue. In the first quarter of 2023, ALDURAZYME net product revenues increased by 41% to $34.4 million compared to the same period in 2022[5].

Market Dynamics

Patient Uptake and Expansion

The growth of ALDURAZYME sales can be attributed to increased patient uptake and expansion into new markets. BioMarin has been successful in increasing awareness and diagnosis of MPS I, leading to more patients being treated with ALDURAZYME.

Competitive Landscape

The market for MPS I treatments is relatively niche, with ALDURAZYME being one of the primary treatments available. However, the landscape is evolving with the introduction of new therapies and gene therapies, which could potentially impact ALDURAZYME's market share in the future.

Geographic Performance

ALDURAZYME sales have been strong across various geographic regions. For example, in the first quarter of 2023, the drug saw significant growth in regions where penetration rates are lower, contributing to overall revenue increase[5].

Financial Trajectory

Revenue Trends

ALDURAZYME has consistently contributed to BioMarin's total enzyme product revenues. In 2023, the drug's revenue growth was part of the overall 15% year-over-year growth in total revenues for BioMarin[5].

Cost and Pricing

The cost of ALDURAZYME is significant due to its complex production process and the rare nature of the disease it treats. Pricing strategies have been crucial in maintaining revenue growth while ensuring patient access.

Research and Development

BioMarin continues to invest in research and development, which includes ongoing studies and potential new indications for ALDURAZYME. This investment is reflected in the company's R&D expenses, which were around 30% of revenue in 2023[5].

Challenges and Opportunities

Generic Competition

While ALDURAZYME is a biologic and less likely to face immediate generic competition, biosimilars could pose a threat in the future. However, the complexity of biologic manufacturing often delays the entry of biosimilars.

Regulatory Environment

Regulatory approvals and updates play a critical role in the sales trajectory of ALDURAZYME. Any changes in regulatory guidelines or the approval of new treatments could impact its market dynamics.

Expansion and New Indications

BioMarin is continually exploring new indications and expanding into new markets, which could further boost ALDURAZYME's sales. For example, the company's efforts to increase awareness and diagnosis in underpenetrated regions can drive growth.

Future Outlook

Growth Projections

BioMarin projects continued growth for its enzyme product revenues, including ALDURAZYME. The company's guidance for 2023 includes a range of $1,700 to $1,850 million for enzyme product revenues, indicating a positive outlook for ALDURAZYME[5].

Market Expansion

The global expansion of ALDURAZYME, particularly in regions with lower penetration rates, is expected to drive future growth. BioMarin's strategic efforts to increase awareness and improve diagnostic capabilities will be key in this expansion.

Innovative Therapies

The emergence of innovative therapies, including gene therapies, presents both opportunities and challenges. While these new therapies could compete with ALDURAZYME, they also highlight the evolving landscape of rare disease treatments and the potential for combination therapies.

Key Takeaways

• Steady Revenue Growth: ALDURAZYME has shown consistent revenue growth since its approval.
• Market Expansion: The drug's sales have been driven by increased patient uptake and geographic expansion.
• Competitive Landscape: ALDURAZYME operates in a niche market but faces potential competition from new therapies.
• Financial Performance: The drug contributes significantly to BioMarin's total enzyme product revenues.
• Future Outlook: Continued growth is projected, driven by market expansion and increased awareness.

FAQs

What is ALDURAZYME used for?

ALDURAZYME is used for the treatment of mucopolysaccharidosis I (MPS I), a rare genetic disorder.

When was ALDURAZYME approved?

ALDURAZYME was approved by the FDA in 2003.

How has ALDURAZYME performed financially in recent years?

In recent years, ALDURAZYME has shown significant revenue growth. For example, in the first quarter of 2023, ALDURAZYME net product revenues increased by 41% to $34.4 million compared to the same period in 2022[5].

What are the main challenges facing ALDURAZYME in the market?

The main challenges include potential competition from new therapies and biosimilars, as well as regulatory changes.

What is the future outlook for ALDURAZYME?

The future outlook is positive, with projected growth driven by market expansion and increased awareness. BioMarin's guidance indicates continued growth for enzyme product revenues, including ALDURAZYME[5].

Sources

1. BioMarin Announces Fourth Quarter and Full Year 2010 Financial Results - BioMarin Pharmaceutical Inc.[2]
2. BioMarin Announces Record Breaking First Quarter 2023 Results - BioMarin Pharmaceutical Inc.[5]
3. Earnings Release-31JUL23 EX99.1 - BioMarin Pharmaceutical Inc.[3]