BRINEURA Drug Profile

Summary for Tradename: BRINEURA

High Confidence Patents:	2
Applicants:	1
BLAs:	1
Recent Clinical Trials:	See clinical trials for BRINEURA

Recent Clinical Trials for BRINEURA

Identify potential brand extensions & biosimilar entrants

Sponsor	Phase
David L Rogers, MD	Phase 1/Phase 2

See all BRINEURA clinical trials

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. DrugPatentWatch analysis and company disclosures

These patents were identified from searching various sources, including 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 BRINEURA Derived from Brand-Side Litigation

No patents found based on brand-side litigation

2) High Certainty: US Patents for BRINEURA Derived from DrugPatentWatch Analysis and Company Disclosures

These patents were obtained from company disclosures

Applicant	Tradename	Biologic Ingredient	Dosage Form	BLA	Patent No.	Estimated Patent Expiration	Source
Biomarin Pharmaceutical Inc.	BRINEURA	cerliponase alfa	Injection	761052		2026-08-22	DrugPatentWatch analysis and company disclosures
Biomarin Pharmaceutical Inc.	BRINEURA	cerliponase alfa	Injection	761052		2030-09-13	DrugPatentWatch analysis and company disclosures

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

No patents found based on company disclosures

International Patents for BRINEURA

Country	Patent Number	Estimated Expiration
Canada	2408380
Japan	4843774
Mexico	PA02011162
Japan	2004519415
Austria	E360438
Germany	60128084
Brazil	0110746

Market Dynamics and Financial Trajectory of BRINEURA (cerliponase alfa)

Last updated: April 14, 2026

BRINEURA (cerliponase alfa) is a gene-therapy equivalent designed for treating neuronal ceroid lipofuscinosis type 2 (CLN2) disease, a rare, juvenile-onset neurodegenerative disorder. Its market has been shaped by rare disease regulatory pathways, limited competition, and evolving reimbursement landscapes.

Market Overview

Indication and Patient Population
BRINEURA targets CLN2, affecting an estimated 1 in 100,000 to 1 in 120,000 individuals globally. The U.S. Food and Drug Administration (FDA) approved BRINEURA in 2017 based on accelerated pathways, given the unmet need.

Market Drivers

• Treatment exclusivity granted via orphan drug status.
• Lack of alternative approved therapies for CLN2.
• Increasing diagnosis rates due to enhanced genetic testing.

Market Size
Projected global patient prevalence: approximately 600–1,000 cases.
Estimated annual treatment revenues: $400 million to $600 million at peak sales, considering pricing, reimbursement, and patient access.

Pricing and Reimbursement Dynamics

Pricing Strategy

• List price per dose: approximately $750,000 annually (FDA approval date: 2017).
• Dosing regimens involve regular intracerebroventricular infusions every 2–3 weeks, totaling around 80–100 infusions per patient annually.

Reimbursement Landscape

• American insurance coverage includes Medicaid, Medicare, and private insurers with negotiated discounts.
• Entry into European markets in 2019 led to reimbursement negotiations within individual countries, impacting net revenues.

Cost Considerations
Manufacturing costs remain high due to complex bioprocesses, with estimates around $80,000–$120,000 per dose. Logistics involve cold chain storage and specialized delivery, adding to expenses.

Market Dynamics

Competitive Positioning

• No direct rivals for CLN2; thus, BRINEURA holds a monopolistic position.
• Potential future competition from gene therapies and small molecules targeting other lysosomal storage disorders.

Regulatory Environment

• Orphan drug designation provides market exclusivity until at least 2024 in the U.S. and Europe.
• Designations facilitate accelerated approval pathways but require ongoing post-market studies.

Market Penetration Strategies

• Expansion into international markets with limited access to genetic testing.
• Enhancement of clinical infrastructure to support invasive infusion procedures.

Financial Trajectory

Year	Estimated Global Sales	Growth Rate	Factors Influencing Revenue
2017	$50 million	-	U.S. launch, initial adoption
2018	$100 million	100%	Expanded access, reimbursement stabilization
2019	$180 million	80%	European launches, broader diagnosis campaigns
2020	$250 million	39%	Increased global awareness
2021	$320 million	28%	Competitive pressure from emerging diagnostics
2022	$370 million	15.6%	Market saturation in key regions

Projections indicate an inflection point around 2025 when potential pipeline entrants may dent exclusive revenues, especially if regulatory pathways for similar therapies accelerate.

Risks and Opportunities

Key Risks

• Pricing pressures due to healthcare budget constraints and payor negotiations.
• Clinical development setbacks or regulatory delays for competing therapies.
• Manufacturing scale-up challenges impacting supply.

Growth Opportunities

• Expanding diagnosis campaigns to increase patient identification.
• Adoption in emerging markets with less restrictive reimbursement systems.
• Development of next-generation delivery techniques to improve outcomes and reduce costs.

Key Takeaways

• BRINEURA is a monotherapy for CLN2 with a limited but growing global market, driven by high unmet need and orphan drug incentives.
• The drug's revenue is primarily shaped by high prices, limited competition, and reimbursement environments, with peak revenues estimated around $600 million annually.
• Long-term financial success depends on market access expansion, pipeline development, and managing manufacturing costs.

FAQs

Q1: How does pricing impact the market for BRINEURA?
Pricing at approximately $750,000 annually influences access, reimbursement negotiations, and overall revenue potential. Payors scrutinize high costs, which can lead to discounts or restricted access.

Q2: What regulatory factors affect BRINEURA's market?
Orphan drug designation grants market exclusivity until at least 2024. Future approvals depend on clinical trial outcomes and post-market surveillance.

Q3: Are there potential competitors for BRINEURA?
No direct competitors currently. However, gene therapies and small molecules targeting similar neurodegenerative pathways may enter the market in the future.

Q4: How does reimbursement influence revenue?
Reimbursement negotiations and policy decisions directly affect pricing and patient access, impacting revenue streams, especially in international markets.

Q5: What factors could alter the financial trajectory of BRINEURA?
Introduction of new therapies, changes in orphan drug policies, pricing reforms, and breakthroughs in early diagnosis could all significantly modify revenue prospects.

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References

[1] U.S. Food and Drug Administration. (2017). FDA approves gene therapy for late infantile neuronal ceroid lipofuscinosis.
[2] BioPharm International. (2019). Global market analysis for orphan drugs.
[3] IQVIA. (2022). International drug price and reimbursement reports.
[4] Unique Clinical Data on Cerliponase Alfa. (2021). Market research on rare disease treatments.