Phenylketonuria Marketed and Pipeline Drugs Assessment, Clinical Trials and Competitive Landscape

• 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.

Scope

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

Reasons to Buy

• 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.