Glycerol phenylbutyrate - Generic Drug Details

What are the generic drug sources for glycerol phenylbutyrate and what is the scope of freedom to operate?

Glycerol phenylbutyrate is the generic ingredient in two branded drugs marketed by Endo Operations and Horizon Therap Us, and is included in two NDAs. There are fifteen patents protecting this compound and one Paragraph IV challenge. Additional information is available in the individual branded drug profile pages.

Glycerol phenylbutyrate has one hundred and fourteen patent family members in twenty-nine countries.

There are two drug master file entries for glycerol phenylbutyrate. One supplier is listed for this compound. There is one tentative approval for this compound.

Summary for glycerol phenylbutyrate

International Patents:	114
US Patents:	15
Tradenames:	2
Applicants:	2
NDAs:	2
Drug Master File Entries:	2
Finished Product Suppliers / Packagers:	1
Raw Ingredient (Bulk) Api Vendors:	33
Clinical Trials:	11
Patent Applications:	110
Patent Litigation and PTAB cases:	See patent lawsuits and PTAB cases for glycerol phenylbutyrate
What excipients (inactive ingredients) are in glycerol phenylbutyrate?	glycerol phenylbutyrate excipients list
DailyMed Link:	glycerol phenylbutyrate at DailyMed

Recent Clinical Trials for glycerol phenylbutyrate

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

Sponsor	Phase
Technical University of Munich	Phase 2
Columbia University	Early Phase 1
Weizmann Institute of Science	Phase 2/Phase 3

See all glycerol phenylbutyrate clinical trials

Generic filers with tentative approvals for GLYCEROL PHENYLBUTYRATE

Applicant	Application No.	Strength	Dosage Form
		1.1G/ML	LIQUID;ORAL

The 'tentative' approval signifies that the product meets all FDA standards for marketing, and, but for the patents / regulatory protections, it would approved.

Pharmacology for glycerol phenylbutyrate

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

Medical Subject Heading (MeSH) Categories for glycerol phenylbutyrate

Cryoprotective Agents

Solvents

Anatomical Therapeutic Chemical (ATC) Classes for glycerol phenylbutyrate

A06AG	Enemas
A06A	DRUGS FOR CONSTIPATION
A06	DRUGS FOR CONSTIPATION
A	Alimentary tract and metabolism

A06AX	Other drugs for constipation
A06A	DRUGS FOR CONSTIPATION
A06	DRUGS FOR CONSTIPATION
A	Alimentary tract and metabolism

A16AX	Various alimentary tract and metabolism products
A16A	OTHER ALIMENTARY TRACT AND METABOLISM PRODUCTS
A16	OTHER ALIMENTARY TRACT AND METABOLISM PRODUCTS
A	Alimentary tract and metabolism

Paragraph IV (Patent) Challenges for GLYCEROL PHENYLBUTYRATE

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 glycerol phenylbutyrate

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		RX	Yes	Yes
Horizon Therap Us	RAVICTI	glycerol phenylbutyrate	LIQUID;ORAL	203284-001	Feb 1, 2013		RX	Yes	Yes
Horizon Therap Us	RAVICTI	glycerol phenylbutyrate	LIQUID;ORAL	203284-001	Feb 1, 2013		RX	Yes	Yes
Horizon Therap Us	RAVICTI	glycerol phenylbutyrate	LIQUID;ORAL	203284-001	Feb 1, 2013		RX	Yes	Yes
Horizon Therap Us	RAVICTI	glycerol phenylbutyrate	LIQUID;ORAL	203284-001	Feb 1, 2013		RX	Yes	Yes
Endo Operations	GLYCEROL PHENYLBUTYRATE	glycerol phenylbutyrate	LIQUID;ORAL	205742-001	Dec 2, 2021		DISCN	No	No

Expired US Patents for glycerol phenylbutyrate

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 glycerol phenylbutyrate

Company	Drugname	Inn	Product Number / Indication	Status	Generic	Biosimilar	Orphan	Marketing Authorisation	Marketing Refusal
Immedica Pharma AB	Ravicti	glycerol phenylbutyrate	EMEA/H/C/003822 Ravicti 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 glycerol phenylbutyrate

Country	Patent Number	Title	Estimated Expiration
Hungary	E040503
South Korea	20190116552	페닐아세트산 전구약물의 치료적 모니터링 방법 (METHODS OF THERAPEUTIC MONITORING OF PHENYLACETIC ACID PRODRUGS)
European Patent Office	2338050	DOSAGE ET SURVEILLANCE DE PATIENTS EN TRAITEMENT PAR DES MÉDICAMENTS DÉSACTIVANT L'AZOTE (DOSING AND MONITORING PATIENTS ON NITROGEN-SCAVENGING DRUGS)
World Intellectual Property Organization (WIPO)	2010025303
European Patent Office	3263102	PROCÉDÉS DE SURVEILLANCE THÉRAPEUTIQUE DE MÉDICAMENTS À RÉCUPÉRATION D'AZOTE (METHODS OF THERAPEUTIC MONITORING OF NITROGEN SCAVENGING DRUGS)
Japan	5577341

Supplementary Protection Certificates for glycerol phenylbutyrate

Patent Number	Supplementary Protection Certificate	SPC Country	SPC Expiration	SPC Description
2330892	2016C/074	Belgium		PRODUCT NAME: GLYCEROLFENYLBUTYRAAT; AUTHORISATION NUMBER AND DATE: EU/1/15/1062 20151201
2330892	132016000126572	Italy		PRODUCT NAME: GLICEROLO FENILBUTIRRATO(RAVICTI); AUTHORISATION NUMBER(S) AND DATE(S): EU/1/15/1062, 20151201
2330892	122016000107	Germany		PRODUCT NAME: GLYCEROLPHENYLBUTYRAT; REGISTRATION NO/DATE: EU/1/15/1062 20151127
2330892	PA2016041,C2330892	Lithuania		PRODUCT NAME: GLICEROLIO FENILBUTIRATAS; REGISTRATION NO/DATE: EU/1/15/1062 20151127
2330892	635	Finland
2330892	C20160044 00204	Estonia		PRODUCT NAME: GLUETSEROOLFENUEUELBUTUERAAT;REG NO/DATE: EU/1/15/1062 01.12.2015

Glycerol phenylbutyrate Market Analysis and Financial Projection Experimental

Market Dynamics and Financial Trajectory for Glycerol Phenylbutyrate

Introduction

Glycerol phenylbutyrate, marketed under the brand name Ravicti, is a crucial medication for the treatment of urea cycle disorders (UCDs). This article delves into the market dynamics and financial trajectory of glycerol phenylbutyrate, highlighting key drivers, restraints, and future projections.

Market Size and Growth

The U.S. urea cycle disorders treatment market, in which glycerol phenylbutyrate is a leading treatment type, is estimated to be valued at US$ 510.0 million in 2023. This market is expected to exhibit a Compound Annual Growth Rate (CAGR) of 3.56% during the forecast period from 2023 to 2030, reaching a projected value of US$ 651.5 million by 2030[1].

Key Drivers

Several factors are driving the growth of the glycerol phenylbutyrate market:

Increasing Product Approvals

Regulatory approvals for new treatments are a significant driver. For instance, the FDA approval of OLPRUVA (sodium phenylbutyrate) in December 2022 for the treatment of certain UCDs has set a positive precedent for similar medications like glycerol phenylbutyrate[1].

Pipeline Products and Collaborations

Manufacturers are focusing on increasing the number of pipeline products for UCDs and collaborating on novel treatments. This includes collaborations between companies like Acer Therapeutics Inc. and Relief Therapeutics Holding AG, which are expected to propel market growth[1].

Inorganic Growth Strategies

Key players are adopting inorganic growth strategies, such as product acquisitions, to strengthen their product portfolios. This strategic approach is expected to boost the market over the forecast period[1].

Market Segmentation

The U.S. urea cycle disorders treatment market is segmented into several categories:

Treatment Type

Glycerol phenylbutyrate dominates the market, accounting for a significant share of 62.8% in 2023. Other treatment types include amino acid supplements, sodium phenylbutyrate, sodium benzoate, and others[1].

Enzyme Deficiency Type

The market is also segmented based on enzyme deficiency types, such as carbamylphosphate synthetase (CPS), ornithine transcarbamylase (OTC), and argininosuccinic acid synthetase (AS) deficiencies[1].

Restraints and Challenges

Despite the growth drivers, there are several challenges affecting the market:

High Cost of Treatment

The high cost of glycerol phenylbutyrate and other UCD treatments is a significant restraint. For example, the annual cost of glycerol phenylbutyrate can be substantially higher than that of sodium phenylbutyrate, with estimates showing a difference of up to £9,241.31 per year in some cases[2].

Patient Non-Compliance

Long-term therapies for UCDs often face issues with patient non-compliance, which can hamper the overall effectiveness and market growth of these treatments[1].

Cost-Effectiveness Analysis

The cost-effectiveness of glycerol phenylbutyrate compared to other treatments, such as sodium phenylbutyrate, is a critical factor. While glycerol phenylbutyrate may offer better control of blood ammonia levels, its cost-effectiveness is often questioned.

• Economic Models: Studies have shown that glycerol phenylbutyrate becomes cost-effective only at high incremental cost-utility ratios (ICURs), often exceeding $1 million per quality-adjusted life-year (QALY) in many scenarios[3].

• Patient Access Schemes: Patient Access Schemes (PAS) can make glycerol phenylbutyrate more cost-effective, but these schemes are often subject to commercial confidentiality and may not be universally applicable[2].

Clinical Efficacy

Glycerol phenylbutyrate has been shown to be non-inferior to sodium phenylbutyrate in controlling blood ammonia levels in patients with UCDs. Clinical trials have supported its efficacy, although long-term data is still being gathered[2][3].

Market Players and Strategies

Key players in the market, such as Horizon Therapeutics Plc, Bausch Health Companies Inc, and Acer Therapeutics Inc., are focusing on strategic collaborations and product acquisitions to strengthen their market positions.

• Acquisitions and Mergers: For example, Amgen's acquisition of Horizon Therapeutics plc in 2023 reflects the industry's trend towards consolidation and expansion into rare disease treatments[4].

Future Projections

The market for glycerol phenylbutyrate is expected to grow steadily, driven by increasing product approvals, pipeline developments, and strategic collaborations among market players.

• Pipeline Developments: The increasing number of pipeline products and novel treatments in development is expected to boost market growth over the forecast period[1].

• Regulatory Support: Continued regulatory support and approvals will be crucial for the market's growth trajectory.

Key Takeaways

• The U.S. urea cycle disorders treatment market, led by glycerol phenylbutyrate, is projected to grow at a CAGR of 3.56% from 2023 to 2030.
• High treatment costs and patient non-compliance are significant restraints.
• Glycerol phenylbutyrate's cost-effectiveness is a critical factor, often requiring Patient Access Schemes to be viable.
• Market growth is driven by product approvals, pipeline developments, and strategic collaborations.

FAQs

Q: What is the current market size of the U.S. urea cycle disorders treatment market? A: The U.S. urea cycle disorders treatment market is estimated to be valued at US$ 510.0 million in 2023[1].

Q: What is the projected CAGR for the U.S. urea cycle disorders treatment market from 2023 to 2030? A: The market is expected to exhibit a CAGR of 3.56% during the forecast period from 2023 to 2030[1].

Q: Which treatment type dominates the U.S. urea cycle disorders treatment market? A: Glycerol phenylbutyrate dominates the market, accounting for a significant share of 62.8% in 2023[1].

Q: What are the major restraints affecting the growth of the glycerol phenylbutyrate market? A: The high cost of treatment and patient non-compliance in long-term therapies are major restraints[1].

Q: How does glycerol phenylbutyrate compare to sodium phenylbutyrate in terms of cost-effectiveness? A: Glycerol phenylbutyrate often has higher ICURs compared to sodium phenylbutyrate, making it less cost-effective in many scenarios unless supported by Patient Access Schemes[2][3].

Cited Sources:

1. Coherent Market Insights: U.S. Urea Cycle Disorders Treatment Market Size By 2030.
2. Scottish Medicines Consortium: Glycerol phenylbutyrate (Ravicti) - SMC advice.
3. NCBI: Glycerol Phenylbutyrate (Ravicti) - Reviewer Worksheets.
4. Amgen Inc.: SHAREHOLDERS 2023.