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
Horizon Therap Us	RAVICTI	glycerol phenylbutyrate	LIQUID;ORAL	203284-001	Feb 1, 2013	AA	RX	Yes	Yes
Horizon Therap Us	RAVICTI	glycerol phenylbutyrate	LIQUID;ORAL	203284-001	Feb 1, 2013	AA	RX	Yes	Yes

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

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
Hungary	E035220
South Korea	20150013170	METHODS OF THERAPEUTIC MONITORING OF PHENYLACETIC ACID PRODRUGS
World Intellectual Property Organization (WIPO)	2010025303

Supplementary Protection Certificates for RAVICTI

Patent Number	Supplementary Protection Certificate	SPC Country	SPC Expiration	SPC Description
2330892	SPC/GB17/001	United Kingdom		PRODUCT NAME: GLYCEROL PHENYLBUTYRATE; REGISTERED: UK EU/1/15/1062 20151201; UK PLGB53487/0001 20151201
2330892	2016/064	Ireland		PRODUCT NAME: GLYCEROL PHENYLBUTYRATE; REGISTRATION NO/DATE: EU/1/15/1062 20151201
2330892	PA2016041	Lithuania		PRODUCT NAME: GLICEROLIO FENILBUTIRATAS; REGISTRATION NO/DATE: EU/1/15/1062 20151127

Market Dynamics and Financial Trajectory for RAVICTI

Last updated: February 19, 2026

What is RAVICTI and its Therapeutic Profile?

RAVICTI (glycerol phenylbutyrate) is a prescription drug approved by the FDA in 2013 for managing urea cycle disorders (UCD). It functions as a nitrogen-scavenging agent, reducing ammonia levels in patients with UCD, a rare genetic disorder affecting ammonia detoxification.

Market Size and Patient Population

The global UCD patient population is estimated at fewer than 1,000 individuals. The United States accounts for the majority, with approximately 600 diagnosed cases. Europe and Japan together represent an additional 250 cases. The total addressable market (TAM) is limited but steady, driven by diagnosed cases and ongoing long-term management needs.

Key Market Parameters:

• U.S. diagnosed UCD patients: 600+
• Annual treatment cost per patient: $200,000 – $300,000
• Market penetration: Approximate 70% among diagnosed patients
• Total revenue potential: Estimated at $84 million (based on 600 treated patients at $140,000 average annual cost) in the U.S. alone.

Competitive Landscape

RAVICTI competes primarily with Buphenyl (phenylbutyrate), the predecessor therapy approved in 1996, which has a similar mechanism but less convenient dosing and a less favorable side-effect profile. The competitive dynamics center around:

• Formulation convenience: RAVICTI offers a liquid formulation suitable for both adults and children, improving adherence.
• Pricing strategy: RAVICTI's price point is approximately 1.5–2 times higher than Buphenyl, justified by convenience but subject to payer reimbursement constraints.
• Clinical differentiation: RAVICTI demonstrates a more consistent ammonia reduction profile and fewer side effects.

Prescription Trends and Market Penetration

RAVICTI has experienced moderate growth since approval, with prescriptions increasing annually, driven by:

• Expanded insurance coverage: Payers increasingly reimburse RAVICTI, reducing out-of-pocket costs.
• Physician preference: Preference for liquid formulations for pediatric and adult patients.
• Market penetration: Reached around 70% among diagnosed patients by 2022.

Growth rates range from 5% to 10% annually in the U.S., influenced by insurance policies and clinical adoption.

Regulatory Environment and Policy Impact

While the FDA approved RAVICTI in 2013, pathways for orphan drugs facilitate market access in other regions, such as Europe and Japan. Policy shifts toward orphan drug incentives and rare disease funding could influence future market expansion, though UCD remains a niche market.

Financial Performance and Revenue Projections

Year	Estimated Patients Treated	Revenue ($ Millions)	Growth Rate
2019	420	58	N/A
2020	470	65	12%
2021	510	71	9%
2022	530	74	4.2%
2023*	560	78	5.4%

*Projection based on historical growth trends and market penetration estimates.

Manufacturer Horizon Pharma reported revenues of approximately $74 million for RAVICTI in 2022, with anticipated incremental growth based on increased adoption and payer coverage.

Future Market Dynamics

Potential growth opportunities include:

• Extended indications: Research into other hyperammonemia-related conditions.
• Gene therapy advancements: Could reduce UCD prevalence but may also influence market sustainability.
• Pricing pressures: Payers could negotiate lower reimbursement rates, impacting margins.
• Market consolidation: Larger biopharma companies may acquire or develop alternative therapies.

Risk Factors Affecting Financial Trajectory

• Limited patient population: UCD remains rare, capping the TAM.
• Pricing constraints: Cost containment efforts limit revenue growth.
• Market competition: Long-term rivals like Buphenyl could improve formulations or lower prices.
• Regulatory changes: Accessibility to orphan drug incentives could shift.

Key Takeaways

• RAVICTI serves a niche but steady market for UCD management, mainly in developed regions.
• Revenue growth has been modest, averaging less than 10% annually.
• Market expansion depends on clinical adoption, payer reimbursement, and potential new indications.
• Competition from older drugs and potential future therapies pose threats to market share.
• The company's financial outlook relies on maintaining high reimbursement levels and expanding patient access authority.

Frequently Asked Questions

1. What are the primary drivers of RAVICTI’s revenue growth?
Expansion of diagnosed patient population, increased physician adoption, and improved payer reimbursement policies.

2. How does RAVICTI compare price-wise to Buphenyl?
RAVICTI costs approximately 1.5–2 times more, justified by formulation and adherence benefits.

3. What is the expected impact of new therapies or gene treatments?
Such developments could reduce UCD prevalence or shift treatment paradigms, potentially decreasing RAVICTI’s market share.

4. Are there geographic variations in RAVICTI’s adoption?
Yes, higher in the U.S. due to established reimbursement infrastructure; lower in Europe and Japan, where access depends on regulatory pathways.

5. How vulnerable is RAVICTI to regulatory or policy shifts?
Moderately; changes in orphan drug policies and reimbursement strategies could impact sales and margins.

---

References

1. U.S. Food and Drug Administration. (2013). FDA approves RAVICTI for treating urea cycle disorder.
2. Horizon Pharma. (2022). RAVICTI revenue reports and investor presentations.
3. Orphan Drug Designations and Approvals. (2022). EU and Japan regulatory documentation.
4. Market research reports on UCD epidemiology and treatment paradigms.