Phenylketonuria Marketed and Pipeline Drugs Assessment, Clinical Trials and Competitive Landscape
Summary
This reports provides a data-driven overview of the current and future competitive landscape in Phenylketonuria therapeutics.
Synopsis
- In 2022, there will be more than 500,000 diagnosed prevalent cases of PKU across 16 pharmaceutical markets.
- There are only two leading marketed drugs for the treatment of PKU, both from BioMarin Pharmaceutical.
- R&D activity in PKU is relatively low, with two products in Phase III development.
- Of the 56 trials investigating PKU that were initiated over the past 10 years, nearly 18% were Phase III trials.
- In deals involving companies with PKU assets, partnerships and licensing agreements are the most common type of deal globally.
- Commercial launch of sepiapterin from PTC Therapeutics is expected in the US and EU in Q4 2023.
GlobalData’s Phenylketonuria: Competitive Landscape combines data from the Pharma Intelligence Center with in-house analyst expertise to provide a competitive assessment of the disease marketplace.
Components of the report include -
- Disease Landscape
- Disease Overview
- Epidemiology Overview
- Treatment Overview
- Marketed Products Assessment
- Breakdown by Mechanism of Action, Molecule Type, Route of Administration
- Product Profiles with Sales Forecast
- Pricing and Reimbursement Assessment
- Retail or Manufacturer Price of Products
- Time to Pricing and Time to Reimbursement
- Pipeline Assessment
- Breakdown by Development Stage, Mechanism of Action, Molecule Type, Route of Administration
- Product Profiles with Sales Forecast
- Late-to-mid-stage Pipeline Drugs
- Phase Transition Success Rate and Likelihood of Approval
- Clinical Trials Assessment
- Breakdown of Trials by Phase, Status, Virtual Components, Sponsors, Geography, and Endpoint Status
- Enrolment Analytics, Site Analytics, Feasibility Analysis
- Deals Landscape
- Mergers, Acquisitions, and Strategic Alliances by Region
- Overview of Recent Deals
- Commercial Assessment
- Key Market Players
- Future Market Catalysts
- Develop and design your in-licensing and out-licensing strategies through a review of pipeline products and technologies, and by identifying the companies with the most robust pipeline.
- Develop business strategies by understanding the trends happening and driving the Phenylketonuria market.
- Drive revenues by understanding the key trends, innovative products and technologies, market segments, and companies likely to impact the global Phenylketonuria market in the future.
- Formulate effective sales and marketing strategies by understanding the competitive landscape and by analyzing the performance of various competitors.
- Identify emerging players with potentially strong product portfolios and create effective counter-strategies to gain a competitive advantage.
- Organize your sales and marketing efforts by identifying the market categories and segments that present maximum opportunities for consolidations, investments, and strategic partnerships.
- Key Findings
- Disease Overview
- Epidemiology Overview
- Epidemiology Overview: Diagnosed Prevalent Cases of PKU in 2022 and 2027
- Treatment Overview
- Treatment Guidelines
- Marketed Drugs Assessment
- Marketed Drugs – Leading Marketed Drugs in PKU
- Marketed Drugs – Overview by Mechanism of Action
- Marketed Drugs – Overview by Route of Administration
- Marketed Drugs – Overview by Molecule Type
- Marketed Drug Profile: BioMarin Pharmaceutical Inc’s Kuvan
- Marketed Drug Profile: BioMarin Pharmaceutical Inc’s Palynziq
- Pricing and Reimbursement Assessment
- Marketed Drugs – Retail Price ($/mg)
- Marketed Drugs – Time to Pricing and Reimbursement for Kuvan
- Marketed Drugs – Time to Pricing and Reimbursement for Palynziq
- Pipeline Drugs Assessment
- Pipeline Drugs Overview – Mid-to-Late-Stage Pipeline Drugs In PKU
- Pipeline Drugs – Overview by Development Stage
- Pipeline Drugs – Overview by Molecule Type
- Pipeline Drugs – Overview by Target
- Pipeline Drugs – Overview by Mechanism of Action
- Pipeline Drugs – Overview by Route of Administration
- Pipeline Drugs – Drug-Specific Phase Transition Success Rate and Likelihood of Approval in PKU
- Pipeline Drugs – Phase Transition Success Rates and Likelihood of Approval in Metabolic Disorders and in PKU
- Clinical Trials Assessment
- Clinical Trials in PKU – Historical Overview
- Clinical Trials in PKU – Overview by Phase
- Clinical Trials in PKU – Overview by Status
- Clinical Trials in PKU – Overview by Phase for Ongoing and Planned Trials
- Clinical Trials in PKU – Trials with a Virtual Component
- Clinical Trials in PKU – Geographic Overview
- Clinical Trials in PKU – Single-Country and Multinational Trials by Region
- Clinical Trials in PKU – Top 20 Sponsors with Breakdown by Phase
- Clinical Trials in PKU – Top 20 Sponsors with Breakdown by Status
- Clinical Trials in PKU – Overview by Endpoint Status
- Clinical Trials in PKU – Overview by Race and Ethnicity
- Clinical Trials in PKU – Enrollment Data
- Clinical Trials in PKU – Overview of Sites by Geography
- Clinical Trials in PKU – Top 20 Countries for Trial Sites
- Clinical Trials in PKU – Top 20 Sites Globally
- Clinical Trials – Feasibility Analysis: Geography Overview
- Clinical Trials – Feasibility Analysis: Benchmark Models for PKU
- Deals Landscape
- Deals Landscape – Mergers, Acquisitions, and Strategic Alliances in PKU by Region
- Commercial Assessment
- Commercial Assessment – Key Market Players in PKU
- Future Market Catalysts – Upcoming Market Catalysts in PKU
- Appendix
- Methodology
- Methodology – Sales Forecasts
- Methodology – Pricing and Reimbursement
- Methodology – Phase Transition Success Rate and Likelihood of Approval Analysis
Research Assistance
Download our eBook: How to Succeed Using Market Research
Learn how to effectively navigate the market research process to help guide your organization on the journey to success.
Download eBook