Sodium phenylbutyrate - Generic Drug Details

What are the generic sources for sodium phenylbutyrate and what is the scope of patent protection?

Sodium phenylbutyrate is the generic ingredient in five branded drugs marketed by Acer, Medunik, Horizon Therap Us, Endo Operations, Sigmapharm Labs Llc, Alvogen, Glenmark Pharms Ltd, and Amylyx, and is included in ten NDAs. There are eight patents protecting this compound. Additional information is available in the individual branded drug profile pages.

Sodium phenylbutyrate has twenty-nine patent family members in twenty-one countries.

There are four drug master file entries for sodium phenylbutyrate. Six suppliers are listed for this compound.

Summary for sodium phenylbutyrate

International Patents:	29
US Patents:	8
Tradenames:	5
Applicants:	8
NDAs:	10
Drug Master File Entries:	4
Finished Product Suppliers / Packagers:	6
Raw Ingredient (Bulk) Api Vendors:	97
Clinical Trials:	39
Patent Applications:	3,608
What excipients (inactive ingredients) are in sodium phenylbutyrate?	sodium phenylbutyrate excipients list
DailyMed Link:	sodium phenylbutyrate at DailyMed

Recent Clinical Trials for sodium phenylbutyrate

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

Sponsor	Phase
Acer Therapeutics Inc.	Phase 2
Jerry Vockley, MD, PhD	Phase 2
Maastricht University	Phase 4

See all sodium phenylbutyrate clinical trials

Pharmacology for sodium phenylbutyrate

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

Anatomical Therapeutic Chemical (ATC) Classes for sodium phenylbutyrate

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

US Patents and Regulatory Information for sodium phenylbutyrate

Applicant	Tradename	Generic Name	Dosage	NDA	Approval Date	TE	Type	RLD	RS	Patent No.	Patent Expiration	Product	Substance	Delist Req.	Exclusivity Expiration
Acer	OLPRUVA	sodium phenylbutyrate	FOR SUSPENSION;ORAL	214860-004	Dec 22, 2022		RX	Yes	Yes	11,154,521		Y
Acer	OLPRUVA	sodium phenylbutyrate	FOR SUSPENSION;ORAL	214860-001	Dec 22, 2022		RX	Yes	No	11,154,521		Y
Medunik	PHEBURANE	sodium phenylbutyrate	PELLETS;ORAL	216513-001	Jun 17, 2022		RX	Yes	Yes
Acer	OLPRUVA	sodium phenylbutyrate	FOR SUSPENSION;ORAL	214860-005	Dec 22, 2022		RX	Yes	No	11,154,521		Y
Amylyx	RELYVRIO	sodium phenylbutyrate; taurursodiol	FOR SUSPENSION;ORAL	216660-001	Sep 29, 2022		DISCN	Yes	No	11,071,742		Y

Expired US Patents for sodium phenylbutyrate

Applicant	Tradename	Generic Name	Dosage	NDA	Approval Date	Patent No.	Patent Expiration
Horizon Therap Us	BUPHENYL	sodium phenylbutyrate	POWDER;ORAL	020573-001	Apr 30, 1996	4,457,942
Horizon Therap Us	BUPHENYL	sodium phenylbutyrate	TABLET;ORAL	020572-001	May 13, 1996	4,457,942

EU/EMA Drug Approvals for sodium phenylbutyrate

Company	Drugname	Inn	Product Number / Indication	Status	Generic	Biosimilar	Orphan	Marketing Authorisation	Marketing Refusal
Eurocept International B. V.	Pheburane	sodium phenylbutyrate	EMEA/H/C/002500 Treatment of chronic management of urea-cycle disorders.	Authorised	no	no	no	2013-07-30
Immedica Pharma AB	Ammonaps	sodium phenylbutyrate	EMEA/H/C/000219 Ammonaps is indicated as adjunctive therapy in the chronic management of urea cycle disorders, involving deficiencies of carbamylphosphate synthetase, ornithine transcarbamylase orargininosuccinate synthetase.It is indicated in all patients with neonatal-onset presentation (complete enzyme deficiencies, presenting within the first 28 days of life). It is also indicated in patients with late-onset disease(partial enzyme deficiencies, presenting after the first month of life) who have a history of hyperammonaemic encephalopathy.	Authorised	no	no	no	1999-12-07

International Patents for sodium phenylbutyrate

Country	Patent Number	Title	Estimated Expiration
Spain	2922749
World Intellectual Property Organization (WIPO)	2017160345
Brazil	112018068665	composições palatáveis incluindo o fenilbutirato de sódio e usos das mesmas
Japan	2022106927	フェニル酪酸ナトリウムを含む口当たりの良い組成物及びその使用
Israel	261777	הרכבים טעימים הכוללים נתרן פנילביוטירייט ושימושים עבורם (Palatable compositions including sodium phenylbutyrate and uses thereof)

Supplementary Protection Certificates for sodium phenylbutyrate

Patent Number	Supplementary Protection Certificate	SPC Country	SPC Expiration	SPC Description
2563920	CR 2019 00001	Denmark		PRODUCT NAME: INOTERSEN AND SALT THEREOF, INCLUDING SODIUM SALTS; REG. NO/DATE: EU/1/18/1296 20180710
1856135	CA 2020 00018	Denmark		PRODUCT NAME: FOSTAMATINIB OR A PHARMACEUTICALLY ACCEPTABLE SALT OF FOSTAMATINIB, OR A HYDRATE, SOLVATE OR N-OXIDE OF FOSTAMATINIB OR THE PHARMACEUTICALLY ACCEPTABLE SALT OF FOSTAMATINIB, ESPECIALLY FOSTAMATINIB DISODIUM, OPTIONALLY IN FORM OF A HYDRATE; REG. NO/DATE: EU/1/19/1405 20200113
2666774	LUC00167	Luxembourg		PRODUCT NAME: RELEBACTAM, EVENTUELLEMENT SOUS FORME DE MONOHYDRATE, IMIPENEME ET CILASTATINE, EVENTUELLEMENT SOUS FORME DE SEL DE SODIUM; AUTHORISATION NUMBER AND DATE: EU/1/19/1420 20200217
2932970	SPC/GB18/041	United Kingdom		PRODUCT NAME: A COMBINATION COMPRISING DOLUTEGRAVIR OR A PHARMACEUTICALLY ACCEPTABLE SALT THEREOF (E.G. DOLUTEGRAVIR SODIUM) AND RILPIVIRINE OR A PHARAMACEUTICALLY ACCEPTABLE SALT THEREOF (E.G. RILPIVIRINE HYDROCHLORIDE); REGISTERED: UK EU/1/18/1282 20180518; UK PLGB 35728/0055 20180518; UK PLGB 35728/0056 20180518; UK PLGB 35728/0057 20180518
1912999	1490062-5	Sweden		PRODUCT NAME: SIMEPREVIR, OR A SALT THEREOF, INCLUDING SIMEPREVIR SODIUM; REG. NO/DATE: EU/1/14/924 20140516

Sodium Phenylbutyrate Market Dynamics and Financial Trajectory

Introduction to Sodium Phenylbutyrate

Sodium phenylbutyrate is a nitrogen-binding drug primarily used to treat urea cycle disorders (UCDs) and maple syrup urine disease (MSUD). These conditions are characterized by the body's inability to break down ammonia, a toxic substance that can accumulate and cause severe health issues.

Market Size and Growth

The global sodium phenylbutyrate market is projected to experience significant growth over the coming years. By 2030, the market is expected to reach approximately $3.02 billion, up from $1.92 billion in 2023, with a Compound Annual Growth Rate (CAGR) of 6.7% during the forecast period[1][4].

Drivers of Market Growth

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

Increasing Incidence of UCDs and MSUD

The rising incidence of urea cycle disorders and maple syrup urine disease is a key driver. According to the Centers for Disease Control and Prevention (CDC), the estimated incidence of UCDs is 1 in 8,500 births[1].

Rising Awareness and Government Support

Growing awareness about the benefits of sodium phenylbutyrate for treating these disorders, along with government support initiatives, is also contributing to market growth. Newborn screening programs, in particular, are helping to identify cases earlier, leading to increased demand for the drug[1].

Advancements in Healthcare Infrastructure

Improving healthcare infrastructure, especially in regions like North America and Europe, is facilitating better diagnosis and treatment options. This infrastructure includes advanced healthcare facilities and increased healthcare expenditure, which are crucial for the market's expansion[1].

Regional Insights

North America

North America is expected to dominate the global sodium phenylbutyrate market due to the presence of major market players and advanced healthcare facilities. The region's growing patient population and robust newborn screening programs further contribute to its market leadership[1].

Europe

Europe is anticipated to be the second-largest market for sodium phenylbutyrate, driven by rising healthcare expenditure and increasing awareness about the treatment options available for UCDs and MSUD[1].

Asia Pacific

The Asia Pacific region is expected to grow at a high rate due to improving healthcare infrastructure, rising medical tourism, and increasing healthcare spending. Key players are investing in research and development to develop enhanced formulations, which will further boost the market in this region[1].

Product Forms and Preferences

The sodium phenylbutyrate market is segmented by product form, with tablets currently dominating the market. However, oral powder formulations are anticipated to witness higher demand in the coming years due to their ease of administration and patient preference[1].

Key Market Players

Several companies are prominent in the sodium phenylbutyrate market, including:

• Horizon Therapeutics plc
• Ammonett Pharma LLC
• Medison Pharma Ltd.
• Modern Dietetics Inc.
• Synageva BioPharma Corp.
• Swedish Orphan Biovitrum AB (Sobi)
• Entera Health, Inc.
• Dipharma Francis S.r.l.
• Ucyclyd Pharma, Inc.

These companies are investing heavily in research and development to enhance their product offerings and gain a competitive edge[1].

Recent Developments and Financial Performance

Amylyx Pharmaceuticals

Amylyx Pharmaceuticals has made significant strides with the approval and commercial launch of RELYVRIO™ (sodium phenylbutyrate and taurursodiol) for the treatment of ALS in adults. The company reported strong financial results, with net product revenue of $102.7 million in the third quarter of 2023, and a total product revenue of $272.3 million in the first three full quarters of the U.S. launch[2][5].

Financial Highlights

• Net Product Revenue: $102.7 million in Q3 2023
• Total Product Revenue: $272.3 million in the first three full quarters of the U.S. launch
• Cash and Short-Term Investments: $355.0 million as of September 30, 2023
• Net Income: $20.9 million in Q3 2023[5]

Challenges and Restraints

Despite the positive growth trajectory, the market faces several challenges:

Stringent Regulatory Approvals

The process of obtaining regulatory approvals is stringent and time-consuming, which can delay the launch of new products and formulations[1].

High R&D Costs

The high costs associated with research and development can be a significant barrier for companies looking to enter or expand in the market[1].

Market Opportunities

Emerging Markets

The Asia Pacific region presents lucrative opportunities due to its improving healthcare infrastructure and increasing healthcare spending. Companies are investing in R&D to develop enhanced formulations tailored to this market[1].

New Product Launches and Partnerships

New product launches, such as RELYVRIO™, and strategic partnerships are expected to drive market growth. Companies are also exploring new applications for sodium phenylbutyrate, such as the treatment of ALS, which opens up additional market opportunities[2][5].

Key Takeaways

• The global sodium phenylbutyrate market is projected to reach $3.02 billion by 2030.
• The market is driven by the increasing incidence of UCDs and MSUD, rising awareness, and government support.
• North America is expected to dominate the market, followed by Europe and the Asia Pacific.
• Key players are investing in R&D to develop enhanced formulations.
• The market faces challenges such as stringent regulatory approvals and high R&D costs.

FAQs

What is sodium phenylbutyrate used for?

Sodium phenylbutyrate is used primarily to treat urea cycle disorders (UCDs) and maple syrup urine disease (MSUD) by binding to nitrogen and helping to remove ammonia from the body.

Which region is expected to dominate the sodium phenylbutyrate market?

North America is expected to dominate the global sodium phenylbutyrate market due to the presence of major market players and advanced healthcare facilities.

What are the key drivers of the sodium phenylbutyrate market?

The key drivers include the increasing incidence of UCDs and MSUD, rising awareness about the benefits of the drug, and government support initiatives.

Which companies are prominent in the sodium phenylbutyrate market?

Companies such as Horizon Therapeutics plc, Ammonett Pharma LLC, Medison Pharma Ltd., and Ucyclyd Pharma, Inc. are prominent players in the market.

What are the challenges faced by the sodium phenylbutyrate market?

The market faces challenges such as stringent regulatory approvals and high research and development costs associated with drug development.

Cited Sources

1. Coherent Market Insights: Sodium Phenylbutyrate Market Size, Trends and Forecast to 2030
2. Amylyx Pharmaceuticals: Third Quarter 2022 Financial Results
3. PR Newswire: The changing dynamics of the Argininosuccinic aciduria market
4. Lucintel: Sodium Phenylbutyrate Market Report: Trends, Forecast and Competitive Analysis
5. Amylyx Pharmaceuticals: Third Quarter 2023 Financial Results

Last updated: 2024-12-17