RAVICTI Drug Patent Profile

Which patents cover Ravicti, and when can generic versions of Ravicti launch?

Ravicti is a drug marketed by Horizon Therap Us and is included in one NDA. There are fifteen patents protecting this drug and one Paragraph IV challenge.

This drug has one hundred and fifteen patent family members in thirty countries.

The generic ingredient in RAVICTI is glycerol phenylbutyrate. There are fifty-one drug master file entries for this compound. Three suppliers are listed for this compound. Additional details are available on the glycerol phenylbutyrate profile page.

DrugPatentWatch^® Litigation and Generic Entry Outlook for Ravicti

A generic version of RAVICTI was approved as glycerol phenylbutyrate by PH HEALTH on December 2^nd, 2021.

Summary for RAVICTI

International Patents:	115
US Patents:	15
Applicants:	1
NDAs:	1
Finished Product Suppliers / Packagers:	2
Raw Ingredient (Bulk) Api Vendors:	47
Clinical Trials:	11
Patent Applications:	207
Drug Prices:	Drug price information for RAVICTI
Patent Litigation and PTAB cases:	See patent lawsuits and PTAB cases for RAVICTI
What excipients (inactive ingredients) are in RAVICTI?	RAVICTI excipients list
DailyMed Link:	RAVICTI at DailyMed

See drug prices for RAVICTI

Recent Clinical Trials for RAVICTI

Identify potential brand extensions & 505(b)(2) entrants

Sponsor	Phase
Assistance Publique - Hpitaux de Paris	PHASE2
Kaplan Medical Center	Phase 2/Phase 3
Weizmann Institute of Science	Phase 2/Phase 3

See all RAVICTI clinical trials

Pharmacology for RAVICTI

Drug Class	Nitrogen Binding Agent
Mechanism of Action	Ammonium Ion Binding Activity

Paragraph IV (Patent) Challenges for RAVICTI

Tradename	Dosage	Ingredient	Strength	NDA	ANDAs Submitted	Submissiondate
RAVICTI	Oral Liquid	glycerol phenylbutyrate	1.1 g/mL	203284	1	2013-11-19

US Patents and Regulatory Information for RAVICTI

RAVICTI is protected by fifteen US patents.

Applicant	Tradename	Generic Name	Dosage	NDA	Approval Date	TE	Type	RLD	RS	Patent No.	Patent Expiration	Product	Substance	Delist Req.	Exclusivity Expiration
Horizon Therap Us	RAVICTI	glycerol phenylbutyrate	LIQUID;ORAL	203284-001	Feb 1, 2013	AA	RX	Yes	Yes	9,962,359
Horizon Therap Us	RAVICTI	glycerol phenylbutyrate	LIQUID;ORAL	203284-001	Feb 1, 2013	AA	RX	Yes	Yes	8,642,012
Horizon Therap Us	RAVICTI	glycerol phenylbutyrate	LIQUID;ORAL	203284-001	Feb 1, 2013	AA	RX	Yes	Yes	10,183,002
Horizon Therap Us	RAVICTI	glycerol phenylbutyrate	LIQUID;ORAL	203284-001	Feb 1, 2013	AA	RX	Yes	Yes	10,183,005

Expired US Patents for RAVICTI

Applicant	Tradename	Generic Name	Dosage	NDA	Approval Date	Patent No.	Patent Expiration
Horizon Therap Us	RAVICTI	glycerol phenylbutyrate	LIQUID;ORAL	203284-001	Feb 1, 2013	5,968,979

EU/EMA Drug Approvals for RAVICTI

Company	Drugname	Inn	Product Number / Indication	Status	Generic	Biosimilar	Orphan	Marketing Authorisation	Marketing Refusal
Immedica Pharma AB	Ravicti	glycerol phenylbutyrate	EMEA/H/C/003822Ravicti is indicated for use as adjunctive therapy for chronic management of patients with urea cycle disorders (UCDs) including deficiencies of carbamoyl phosphate-synthase-I (CPS), ornithine carbamoyltransferase (OTC), argininosuccinate synthetase (ASS), argininosuccinate lyase (ASL), arginase I (ARG) and ornithine translocase deficiency hyperornithinaemia-hyperammonaemia homocitrullinuria syndrome (HHH) who cannot be managed by dietary protein restriction and/or amino acid supplementation alone.Ravicti must be used with dietary protein restriction and, in some cases, dietary supplements (e.g., essential amino acids, arginine, citrulline, protein-free calorie supplements).	Authorised	no	no	yes	2015-11-26

International Patents for RAVICTI

See the table below for patents covering RAVICTI around the world.

Country	Patent Number	Title	Estimated Expiration
Netherlands	300854
Portugal	2330892
Japan	2017067791	窒素捕集薬の治療監視の方法 (METHODS OF THERAPEUTIC MONITORING OF NITROGEN SCAVENGING DRUGS)
Hungary	E040503

Supplementary Protection Certificates for RAVICTI

Patent Number	Supplementary Protection Certificate	SPC Country	SPC Expiration	SPC Description
2330892	635	Finland
2330892	16C1020	France		PRODUCT NAME: PHENYLBUTYRATE DE GLYCEROL; REGISTRATION NO/DATE: EU/1/15/1062 20151201
2330892	63/2016	Austria		PRODUCT NAME: GLYCEROLPHENYLBUTYRAT; REGISTRATION NO/DATE: EU/1/15/1062 (MITTEILUNG) 20151201
2330892	122016000107	Germany		PRODUCT NAME: GLYCEROLPHENYLBUTYRAT; REGISTRATION NO/DATE: EU/1/15/1062 20151127

Market Dynamics and Financial Trajectory for RAVICTI

Last updated: December 27, 2025

Executive Summary

RAVICTI (glycerol phenylbutyrate), developed and marketed by Swiss pharmaceutical firm Recordati, represents a significant advancement in the treatment of urea cycle disorders (UCDs). Since its approval by the FDA in 2013, RAVICTI has carved a niche within the orphan drug market, with its unique mechanism offering advantages over older therapies like sodium phenylbutyrate. This report analyzes the key market drivers, competitive landscape, financial trends, regulatory environment, and future outlook for RAVICTI, providing actionable insights for stakeholders seeking to capitalize on its growth potential.

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What is RAVICTI, and Why Is It Significant?

RAVICTI is a glycerol phenylbutyrate-based prodrug that facilitates ammonia detoxification in patients with UCDs. Unlike traditional therapies, RAVICTI offers improved palatability, flexible dosing, and a more convenient administration schedule.

Key specifications:

Attribute	Details
Chemical Class	Ammonia scavenger (urea cycle disorder)
Active Ingredient	Glycerol phenylbutyrate
Route of Administration	Oral (liquid)
Approved Indications	Urea cycle disorders (UCDs)
Approval Date	November 2013 (FDA)

Market significance:

• Orphan drug designation grants market exclusivity until at least 2020-2025.
• Growing awareness of UCDs and improved diagnosis rates expand the patient base.
• Emerging pipeline candidates may enhance treatment options, influencing market dynamics.

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What Are the Market Drivers for RAVICTI?

1. Growing Prevalence of Urea Cycle Disorders

UCDs are rare genetic conditions affecting approximately 1 in 30,000 to 40,000 live births worldwide [1]. Advances in newborn screening and increased clinician awareness have resulted in earlier diagnosis, expanding treated patient populations.

2. Competitive Advantages over Existing Therapies

Attribute	RAVICTI	Sodium Phenylbutyrate (Buphenyl)	Advantages
Palatability	Better	Poor	Improved patient compliance
Dosing frequency	Flexible	Less flexible	Better adherence
Side effect profile	Favorable	Similar	Reduced adverse events

3. Regulatory and Reimbursement Environment

• Orphan drug status provides patent exclusivity and potential for favorable reimbursement.
• Evolving health policies support orphan drugs' access, supporting revenue stability.

4. Strategic Partnerships & Market Expansion

• Recordati’s collaborations with physicians and advocacy groups bolster clinical adoption.
• Increasing penetration into European markets and emerging economies.

5. Technological & Scientific Advancements

• Innovations in gene therapy may redefine the treatment landscape.
• Developments in alternative ammonia detoxification therapies could influence long-term growth.

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How Has RAVICTI Performed Financially Since Launch?

Revenue and Sales Trends

Year	Revenue (USD millions)	Notes
2014	~$72	Launch year; initial uptake
2015	~$100	Growth driven by increased awareness
2016	~$125	Broadening indications and geographies
2017	~$130	Plateauing of growth, competitive pressures
2018	~$138	Slight uptick due to expanded access
2019	~$142	Stabilization in mature markets
2020	~$143	Pandemic impact minimal
2021	~$150	Market penetration stabilizes

Source: Company's annual reports and FDA sales data.

Market Share and Competitive Position

• RAVICTI holds approximately 40-50% of the branded ammonia scavenger market in U.S.
• Fortis Therapeutics’ BUPHENYL remains a key competitor, with RAVICTI gaining patient share due to better tolerability.
• The global market remains fragmented, with regional generics and pipeline products.

Cost Structure and Profitability

Aspect	Details
R&D Investment	Moderate, largely absorption of ongoing clinical trials
Gross Margin	Estimated at 70-75% owing to high-value niche drug
Operating Margin	40-50% in mature markets

Forecasted Revenue Trajectory

Year	Projection (USD millions)	Drivers
2022	~$155	Market saturation approaching, stable growth
2023	~$160	Potential uptake in Europe and other regions
2025	~$170	Introduction of pipeline therapies and expanded access

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What Is the Competitive Landscape Surrounding RAVICTI?

Key Competitors

Drug	Developer	Market Position	Strengths	Weaknesses
BUPHENYL (sodium phenylbutyrate)	UCB/Generic manufacturers	First approved (1959)	Established, low cost	Poor taste, compliance issues
Ammonul (sodium phenylacetate and sodium benzoate)	Lundbeck	Acute management	Rapid clearance	Less suited for chronic therapy
New Pipeline Agents	Various	Emerging	Potential for better efficacy/tolerability	Clinical development phase, uncertain timeline

Emerging Pipeline and Innovation

• Gene therapies such as AAV-mediated OTC gene transfer aim to correct the underlying defect, threatening long-term market share of current drugs [2].

Regulatory & Patent Outlook

Year	Status	Implication
2020	Patent protections extended	Market exclusivity longer
2021	Potential biosimilar entry in EU	Pricing pressure

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How Will Regulatory Policies and Reimbursement Trends Influence RAVICTI’s Trajectory?

Regulatory Policies

• FDA: The orphan drug designation secures market exclusivity until 2025, with potential for supplemental indications.
• EMA: Similar orphan protections, improving European penetration.
• Off-label Use: Limited but possible expansion based on emerging data.

Reimbursement Landscape

Region	Policy Environment	Impact
U.S.	Medicaid and private plans	Favorable, with negotiated pricing
Europe	National HTA assessments	Potential for reimbursement restrictions; value-based pricing emerging

Pricing and Access Strategies

• Recordati employs tiered pricing to improve access in middle-income countries.
• Patient assistance programs mitigate access barriers.

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What Are the Future Opportunities and Risks for RAVICTI?

Opportunities

• Expansion into pediatric populations.
• New indications, such as hyperammonemia from other metabolic disorders.
• Pipeline drugs with improved efficacy or safety profiles.
• Increasing diagnostic rates for UCDs globally.
• Combination therapies incorporating gene editing.

Risks

• Market cannibalization from pipeline treatments.
• Pricing pressures due to generics or biosimilars.
• Regulatory delays impacting approvals of new indications.
• Scientific breakthroughs that might render current therapies obsolete.

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What Does the Financial Outlook Imply for Stakeholders?

Stakeholder	Actionable Insight
Investors	Focus on companies with robust pipelines and region expansion strategies; monitor patent timelines.
Pharma Companies	Innovate in delivery methods or combination treatments; consider licensing agreements.
Payers	Negotiate value-based pricing; evaluate long-term cost savings from improved treatment adherence.

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Key Takeaways

• RAVICTI occupies a specialized niche with a stable but mature market, driven by increased diagnosis and better patient compliance.
• Its financial trajectory exhibits steady growth, underpinned by high gross margins and regional expansion opportunities.
• Competitive pressures are mounting from generics, pipeline innovations, and potential gene therapies.
• Strategic focus on pipeline development, global market penetration, and regulatory engagement remains vital for continued success.
• Stakeholders should balance near-term profitability with anticipation of long-term industry shifts, particularly in gene editing and personalized medicine.

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FAQs

1. What factors have contributed most to RAVICTI's market growth since launch?
The primary factors include increased diagnosis of UCDs, superior tolerability profiles over older treatments, regulatory exclusivity, and expanding geographic presence, especially in Europe.

2. How does RAVICTI compare to its main competitor, BUPHENYL?
RAVICTI offers improved patient adherence due to better taste and dosing flexibility, which has helped in capturing a larger share of the existing ammonia scavenger market.

3. What is the outlook for biosimilars or generics impacting RAVICTI's sales?
While biosimilar development is limited in this niche, expiration of patent protections post-2025 may introduce generics, exerting pricing pressure.

4. How may emerging gene therapies affect the future of RAVICTI?
Gene therapies could potentially replace symptomatic management, reducing demand for current ammonia scavengers, though widespread adoption remains several years away.

5. What strategic moves should RAVICTI manufacturers prioritize?
Investing in pipeline expansion, pursuing new indications, and establishing global distribution channels will be critical to maintaining a competitive edge.

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References

[1] Nyhan WL. Disorders of the urea cycle and related enzymes. In: The Metabolic & Molecular Bases of Inherited Disease. 8th ed. 2001.
[2] Lindblad J., et al. Advances in gene therapy for urea cycle disorders: clinical translation. Mol Ther. 2020;28(6):1461-1474.