CLINICAL TRIALS PROFILE FOR RAVICTI

All Clinical Trials for Ravicti

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT01257737 ↗	To Evaluate the Safety of Long-term Use of HPN-100 in the Management of Urea Cycle Disorders (UCDs)	Completed	Horizon Therapeutics, LLC	Phase 4	2010-10-04	This was an open-label, long-term safety study of HPN-100 (RAVICTI; glycerol phenylbutyrate) in participants with a urea cycle disorder (UCD) who completed the safety extensions of HPN-100-005 (NCT00947544; HPN-100-005SE), HPN-100-006 (NCT00947297; HPN-100-007), or HPN-100-012 (NCT01347073; HPN-100-012SE). The initial studies were 1- to 2-week crossover studies, and their associated safety extensions were 12-month, open-label studies. All participants who completed the initial studies were eligible to enroll in the associated safety extension studies, and new participants were also permitted to enroll directly into the safety extension studies.
NCT01347073 ↗	Study of the Safety, Pharmacokinetics and Efficacy of HPN-100, in Pediatric Subjects With Urea Cycle Disorders (UCDs)	Completed	Horizon Pharma Ireland, Ltd., Dublin Ireland	Phase 3	2011-07-01	This non-randomized, open-label study was approximately one year in duration and consisted of a short term NaPBA to HPN-100 switchover part involving two overnight stays followed by a 12-month long term treatment period involving monthly visits.
NCT01881984 ↗	Use of Ravicti™ in Patients With MCAD Deficiency With the 985A>G (K304E) Mutation	Completed	Horizon Pharma Ireland, Ltd., Dublin Ireland	Phase 1	2013-06-01	This is a medical research study to test a medication in adult patients with a disease called medium-chain acyl-CoA dehydrogenase (MCAD) deficiency caused by at least one copy of the 985A>G mutation. The medication is glycerol phenylbutyrate, called Ravicti, which is currently FDA approved for the treatment of urea cycle disorders. Previous research suggests that Ravicti may also be effective in the treatment MCAD deficiency. This study will investigate the safety and efficacy (how well it works) of Ravicti in patients with MCAD deficiency caused by having at least one copy of the 985A>G mutation.
NCT01881984 ↗	Use of Ravicti™ in Patients With MCAD Deficiency With the 985A>G (K304E) Mutation	Completed	University of Pittsburgh	Phase 1	2013-06-01	This is a medical research study to test a medication in adult patients with a disease called medium-chain acyl-CoA dehydrogenase (MCAD) deficiency caused by at least one copy of the 985A>G mutation. The medication is glycerol phenylbutyrate, called Ravicti, which is currently FDA approved for the treatment of urea cycle disorders. Previous research suggests that Ravicti may also be effective in the treatment MCAD deficiency. This study will investigate the safety and efficacy (how well it works) of Ravicti in patients with MCAD deficiency caused by having at least one copy of the 985A>G mutation.
NCT01949766 ↗	Transition From Buphenyl to RAVICTI for the Therapy of Byler Disease	No longer available	University of Pittsburgh		1969-12-31	This is a single patient compassionate use protocol to determine whether RAVICTI will improve bile flow in a subject who previously tolerated therapy with Buphenyl.

Clinical Trial Conditions for Ravicti

Condition Name

Condition Name for Ravicti
Intervention	Trials
Urea Cycle Disorder	2
Urea Cycle Disorders	2
Byler Disease	2
Cystic Fibrosis	1

Condition MeSH

Condition MeSH for Ravicti
Intervention	Trials
Disease	4
Urea Cycle Disorders, Inborn	4
Cholestasis, Intrahepatic	2
Fibrosis	1

Clinical Trial Locations for Ravicti

Trials by Country

Trials by Country for Ravicti
Location	Trials
United States	47
Spain	3
Italy	2
Canada	2
Israel	1

Trials by US State

Trials by US State for Ravicti
Location	Trials
Pennsylvania	5
Ohio	4
New York	4
Colorado	3
California	3

Clinical Trial Progress for Ravicti

Clinical Trial Phase

Clinical Trial Phase for Ravicti
Clinical Trial Phase	Trials
Phase 4	3
Phase 3	1
Phase 2/Phase 3	1

Clinical Trial Status

Clinical Trial Status for Ravicti
Clinical Trial Phase	Trials
Completed	4
Recruiting	3
No longer available	2

Clinical Trial Sponsors for Ravicti

Sponsor Name

Sponsor Name for Ravicti
Sponsor	Trials
Horizon Therapeutics, LLC	3
Horizon Pharma Ireland, Ltd., Dublin Ireland	3
University of Pittsburgh	3

Sponsor Type

Sponsor Type for Ravicti
Sponsor	Trials
Other	12
Industry	6

Ravicti: Clinical Trials, Market Analysis, and Projections

Introduction to Ravicti

Ravicti, also known as glycerol phenylbutyrate, is a medication used to treat urea cycle disorders (UCDs) and other conditions characterized by elevated ammonia levels in the blood. Here, we will delve into the current clinical trials, market analysis, and future projections for Ravicti.

Clinical Trials Update

Ongoing and Recent Trials

Several clinical trials are ongoing or have recently concluded to evaluate the efficacy and safety of Ravicti in various patient populations.

Trial for Children with Creatine Transporter Deficiency

A notable clinical trial has been expanded to include children with Creatine Transporter Deficiency (SLC6A8 mutations) who experience seizures. This open-label study aims to evaluate the use of glycerol phenylbutyrate for monogenetic developmental epileptic encephalopathies (DEEs), characterized by epilepsy and developmental delay in early life. Interested participants must attend an informational webinar to express their interest in participating[1].

Safety, Efficacy, and Pharmacokinetics in Pediatric Subjects

Another open-label study focuses on the safety, efficacy, and pharmacokinetics of Ravicti in pediatric subjects under 2 years of age with UCDs. This study includes a transition period to Ravicti followed by a safety extension period of at least 6 months and up to 24 months. It aims to capture critical information on safety, pharmacokinetics, and efficacy in young children with UCDs[4].

Trial Outcomes and Efficacy

Previous Phase III studies have demonstrated the efficacy of Ravicti. For instance, a Phase III study achieved its primary efficacy endpoint, showing non-inferiority of Ravicti to sodium phenylbutyrate (NaPBA) in controlling blood ammonia levels in patients with UCDs[3].

Market Analysis

Current Market Size and Growth

The global Ravicti market is experiencing significant growth driven by increasing awareness of UCDs and advancements in rare disease research.

• As of 2024, the global Ravicti market size is estimated to be USD 105.20 million[2].
• The market is projected to expand at a compound annual growth rate (CAGR) of 26.20% from 2024 to 2031, reaching USD 536.33 million by 2031[2].

Regional Market Performance

The market performance varies by region:

• North America: Expected to contribute significantly, with a market size of USD 42.08 million in 2024 and a CAGR of 24.4% from 2024 to 2031[2].
• Asia Pacific: Holds around 23% of the global revenue, with a market size of USD 24.20 million in 2024 and a CAGR of 28.2% from 2024 to 2031[2].
• Latin America and Middle East & Africa: Smaller but growing markets, with CAGRs of 25.6% and 25.9%, respectively[2].

Market Drivers and Trends

Key drivers of the Ravicti market include:

• Increasing Awareness of UCDs: Higher diagnosis rates and demand for effective treatments are driving market growth[2].
• Advancements in Rare Disease Research: Growing investment in research is advancing therapeutic options and improving patient outcomes[2].
• Personalized Medicine and Targeted Therapies: A heightened focus on personalized medicine aligns with the specialized nature of Ravicti[2].
• Orphan Drug Incentives: Regulatory support for rare diseases further supports market expansion[2].

Challenges

Despite the positive trends, the market faces challenges such as:

• High Treatment Costs: A significant barrier to access for many patients[2].
• Potential Disruptions: Events like the COVID-19 pandemic can impact market dynamics[2].

Market Projections

Forecasted Market Size

Different reports provide slightly varying projections, but all indicate substantial growth:

• According to Cognitive Market Research, the global Ravicti market is expected to reach USD 536.33 million by 2031, with a CAGR of 26.20% from 2024 to 2031[2].
• Valuates Reports estimate the market to reach USD 556.5 million by 2030, with a CAGR of 6.8% during the forecast period 2024-2030[5].

Dominant Market Segments

The 25ml formulation of Ravicti is expected to dominate the market due to its convenience, suitability for a broad range of patients, and alignment with standard treatment protocols. This dosage allows for easier administration and dosage adjustments, making it a preferred option for healthcare providers[2].

Key Players

The main player in the Ravicti market is Horizon Pharma, which is responsible for the development and distribution of Ravicti. The company's efforts in research, marketing, and regulatory compliance are crucial for the drug's market performance[5].

Conclusion

Ravicti is a critical medication for patients with urea cycle disorders and other conditions involving elevated ammonia levels. The ongoing clinical trials and expanding patient populations indicate a strong potential for continued growth.

Key Takeaways

• Clinical Trials: Ongoing trials are evaluating Ravicti's efficacy and safety in children with Creatine Transporter Deficiency and pediatric subjects under 2 years of age with UCDs.
• Market Growth: The global Ravicti market is projected to grow significantly, driven by increasing awareness of UCDs and advancements in rare disease research.
• Regional Performance: North America, Asia Pacific, and other regions are expected to contribute to the market growth, each with their own CAGRs.
• Market Drivers: Increasing awareness, research advancements, and regulatory support are key drivers of the market.
• Challenges: High treatment costs and potential disruptions from events like the COVID-19 pandemic are challenges to be addressed.

FAQs

What is Ravicti used for?

Ravicti, or glycerol phenylbutyrate, is used to treat urea cycle disorders (UCDs) and other conditions characterized by elevated ammonia levels in the blood.

What are the current clinical trials for Ravicti?

Current trials include an open-label study for children with Creatine Transporter Deficiency and a study on the safety, efficacy, and pharmacokinetics in pediatric subjects under 2 years of age with UCDs.

What is the projected market size for Ravicti by 2031?

The global Ravicti market is expected to reach USD 536.33 million by 2031, with a CAGR of 26.20% from 2024 to 2031[2].

Who is the main player in the Ravicti market?

The main player in the Ravicti market is Horizon Pharma.

What are the key drivers of the Ravicti market?

Key drivers include increasing awareness of UCDs, advancements in rare disease research, a focus on personalized medicine, and regulatory support for rare diseases.

Sources

1. Association for Creatine Deficiencies, "Ravicti Clinical Trial Announcement," Ravicti Clinical Trial - Association for Creatine Deficiencies.
2. Cognitive Market Research, "Global Ravicti Market Report 2024," The global Ravicti market size will be USD 105.20 million in 2024.
3. Health Canada, "Regulatory Decision Summary for RAVICTI," Regulatory Decision Summary for RAVICTI.
4. Stanford Medicine, "A Study of the Safety, Efficacy and Pharmacokinetics of Glycerol Phenylbutyrate in Pediatric Subjects Under 2 Years of Age With Urea Cycle Disorders," A Study of the Safety, Efficacy and Pharmacokinetics of Glycerol ....
5. Valuates Reports, "Global Ravicti Market Research Report 2024," Global Ravicti Market Research Report 2024.