Sodium phenylbutyrate; taurursodiol - Generic Drug Details

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

Sodium phenylbutyrate; taurursodiol is the generic ingredient in one branded drug marketed by Amylyx and is included in one NDA. There are five patents protecting this compound. Additional information is available in the individual branded drug profile pages.

Sodium phenylbutyrate; taurursodiol has fifty-four patent family members in twenty-four countries.

One supplier is listed for this compound.

Summary for sodium phenylbutyrate; taurursodiol

International Patents:	54
US Patents:	5
Tradenames:	1
Applicants:	1
NDAs:	1
Finished Product Suppliers / Packagers:	1
DailyMed Link:	sodium phenylbutyrate; taurursodiol at DailyMed

DrugPatentWatch^® Estimated Loss of Exclusivity (LOE) Date for sodium phenylbutyrate; taurursodiol

Constraining patent/regulatory exclusivity:

TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS) IN ADULTS

Dosage:

FOR SUSPENSION;ORAL

^*The generic entry opportunity date is the latter of the last compound-claiming patent and the last regulatory exclusivity protection. Many factors can influence early or later generic entry. This date is provided as a rough estimate of generic entry potential and should not be used as an independent source.

Pharmacology for sodium phenylbutyrate; taurursodiol

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

Anatomical Therapeutic Chemical (ATC) Classes for sodium phenylbutyrate; taurursodiol

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; taurursodiol

Applicant	Tradename	Generic Name	Dosage	NDA	Approval Date	TE	Type	RLD	RS	Patent No.	Patent Expiration	Product	Substance	Delist Req.	Exclusivity Expiration
Amylyx	RELYVRIO	sodium phenylbutyrate; taurursodiol	FOR SUSPENSION;ORAL	216660-001	Sep 29, 2022		DISCN	Yes	No
Amylyx	RELYVRIO	sodium phenylbutyrate; taurursodiol	FOR SUSPENSION;ORAL	216660-001	Sep 29, 2022		DISCN	Yes	No
Amylyx	RELYVRIO	sodium phenylbutyrate; taurursodiol	FOR SUSPENSION;ORAL	216660-001	Sep 29, 2022		DISCN	Yes	No

International Patents for sodium phenylbutyrate; taurursodiol

Country	Patent Number	Title	Estimated Expiration
China	110787169	用于提高细胞存活力的组合物和使用该组合物的方法 (Compositions for improving cell viability and methods of use thereof)
European Patent Office	4076426
Japan	7080944

Supplementary Protection Certificates for sodium phenylbutyrate; taurursodiol

Patent Number	Supplementary Protection Certificate	SPC Country	SPC Expiration	SPC Description
0290047	SPC/GB97/078	United Kingdom		PRODUCT NAME: MANGAFODIPIR AND PHARMACEUTICALLY ACCEPTABLE SALTS THEREOF, IN PARTICULAR MANGAFODIPIR TRISODIUM; REGISTERED: UK EU/1/97/040/001 19970522; UK EU/1/97/040/002 19970522
2203431	15C0013	France		PRODUCT NAME: DASABUVIR OU UN SEL DE CELUI-CI, NOTAMMENT LE SEL DE SODIUM; REGISTRATION NO/DATE: EU/1/14/983 20150119
0480717	98C0022	France		PRODUCT NAME: MONTELUKAST SODIUM; REGISTRATION NO/DATE IN FRANCE: NL 23 133 DU 19980320; REGISTRATION NO/DATE AT EEC: 13 651 DU 19970825

Sodium phenylbutyrate; taurursodiol Market Analysis and Financial Projection Experimental

Market Dynamics and Financial Trajectory for Sodium Phenylbutyrate and Taurursodiol

Introduction

Sodium phenylbutyrate and taurursodiol, combined as AMX0035 (RELYVRIO), have emerged as significant players in the treatment of urea cycle disorders (UCDs) and amyotrophic lateral sclerosis (ALS). Here, we delve into the market dynamics and financial trajectory of these drugs.

Market Size and Growth

The global sodium phenylbutyrate market is projected to grow substantially, driven by increasing incidence of UCDs and ALS. By 2030, the market is expected to reach $3.02 billion from $1.92 billion in 2023, at a Compound Annual Growth Rate (CAGR) of 6.7%[1].

Key Drivers

Increasing Incidence of UCDs and ALS

The growth of the sodium phenylbutyrate market is significantly driven by the rising incidence of UCDs and ALS. UCDs affect approximately 1 in 8,500 births, while ALS, a fatal neurodegenerative disease, has seen increased awareness and demand for effective treatments[1][3].

Regulatory Approvals

Recent regulatory approvals have been a major boost. For instance, the FDA approval of RELYVRIO (sodium phenylbutyrate and taurursodiol) for the treatment of adults with ALS in September 2022 has opened new avenues for market growth[2][4].

Government Support and Awareness

Government support initiatives and rising awareness about the benefits of sodium phenylbutyrate for treating UCDs and ALS are also driving market expansion. Newborn screening programs and increased healthcare expenditure in regions like North America and Europe further contribute to this growth[1][3].

Market Segments

Regional Insights

• North America: This region is expected to dominate the global sodium phenylbutyrate market due to the presence of major market players, advanced healthcare facilities, and high incidence of UCDs and ALS[1][3].
• Europe: Europe is anticipated to be the second-largest market, driven by rising healthcare expenditure and a strong focus on research and development[1].
• Asia Pacific: The market in this region is expected to grow at a high rate due to improving healthcare infrastructure, rising medical tourism, and increasing healthcare spending[1].

Distribution Channels

• Hospital Pharmacies: These currently account for a notable share of the market, followed by retail pharmacies. The online segment is expected to expand significantly due to increasing internet penetration and government initiatives[3].
• Oral Segment: The oral segment, including tablets and oral powders, dominates the market. However, oral powder formulations are anticipated to witness higher demand in the coming years[1].

Financial Trajectory

Revenue and Expenses

• Amylyx Pharmaceuticals: For the third quarter of 2022, Amylyx Pharmaceuticals reported net product revenue of $0.3 million, primarily from sales of ALBRIOZA in Canada. Research and development expenses were $24.9 million, and selling, general, and administrative expenses were $29.9 million. The net loss for the quarter was $53.8 million[2].
• Market Projections: The amyotrophic lateral sclerosis treatment market, which includes RELYVRIO, is projected to reach $1.038 billion by 2032 from $662.3 million in 2022, growing at a CAGR of 4.6%[4].

Pricing and Affordability

• Cost of Treatment: AMX0035, the combination of sodium phenylbutyrate and taurursodiol, currently costs $158,000 annually in the US, which may pose a financial barrier for many patients[5].

Challenges and Opportunities

Regulatory Hurdles

• Stringent regulatory approvals and high research and development costs associated with drug development can hamper market growth. However, recent approvals and ongoing clinical trials (such as the phase 3 PHOENIX trial) are expected to mitigate these challenges[1][2].

Side Effects and Safety

• Common side effects of sodium phenylbutyrate, such as missed menstrual periods, loss of appetite, headache, nausea, and lower back pain, may affect market growth. However, the safety and efficacy of AMX0035 have been demonstrated in clinical trials, including the CENTAUR study and its open-label extension[3][5].

Research and Development

• Key players are investing heavily in R&D to develop enhanced formulations and expand their market share. For example, Amylyx Pharmaceuticals is conducting IND-enabling studies for AMX0114, a new compound targeting Calpain-2[2].

Key Players

• Horizon Therapeutics plc
• Ammonett Pharma LLC
• Medison Pharma Ltd.
• Amylyx Pharmaceuticals, Inc.
• Swedish Orphan Biovitrum AB (Sobi)
• Entera Health, Inc.
• Dipharma Francis S.r.l.
• Ucyclyd Pharma, Inc.

These companies are actively involved in the production, distribution, and sale of sodium phenylbutyrate and are driving market growth through innovative products and strategic partnerships[1].

Recent Developments

• FDA Approval: The FDA approval of RELYVRIO in September 2022 marked a significant milestone for the treatment of ALS[2][4].
• Clinical Trials: Ongoing and planned clinical trials, such as the phase 3 PHOENIX trial, are expected to provide further insights into the safety and efficacy of AMX0035[2][5].

Market Trends

• Oral Formulations: The oral segment, particularly oral powders, is expected to witness higher demand due to ease of administration and patient compliance[1].
• Online Pharmacies: The online segment is expanding rapidly due to increased internet penetration and government initiatives to improve healthcare access[3].

Key Takeaways

• The global sodium phenylbutyrate market is projected to grow significantly, driven by increasing incidence of UCDs and ALS.
• Regulatory approvals, government support, and rising awareness are key drivers of market growth.
• North America dominates the market, followed by Europe and Asia Pacific.
• The oral segment, particularly oral powders, is expected to grow in demand.
• High research and development costs and side effects are potential challenges.
• Key players are investing in R&D to develop enhanced formulations.

FAQs

What is the projected market size of sodium phenylbutyrate by 2030?

The global sodium phenylbutyrate market is projected to reach $3.02 billion by 2030[1].

What are the primary uses of sodium phenylbutyrate?

Sodium phenylbutyrate is primarily used to treat urea cycle disorders (UCDs) and amyotrophic lateral sclerosis (ALS)[1][4].

Which region dominates the sodium phenylbutyrate market?

North America dominates the global sodium phenylbutyrate market due to the presence of major market players and advanced healthcare facilities[1][3].

What is the cost of AMX0035 (RELYVRIO) annually in the US?

AMX0035 currently costs $158,000 annually in the US, which may pose a financial barrier for many patients[5].

What are the common side effects of sodium phenylbutyrate?

Common side effects include missed menstrual periods, loss of appetite, headache, nausea, and lower back pain[3].

Sources

1. Coherent Market Insights: Sodium Phenylbutyrate Market Size, Trends and Forecast to 2030.
2. Amylyx Pharmaceuticals: Amylyx Pharmaceuticals Reports Third Quarter 2022 Financial Results.
3. BioSpace: Sodium Phenylbutyrate Drugs Market: Oral Segment Witness Highest Growth.
4. Allied Market Research: Amyotrophic Lateral Sclerosis Treatment Market Forecast - 2032.
5. PubMed: An evaluation of the combination of sodium phenylbutyrate and taurursodiol.