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

Publisher GlobalData
Published Sep 28, 2022
Length 69 Pages
SKU # GBDT17487297

Description

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.
Scope

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
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.

Table of Contents

69 Pages
  • Key Findings
  • Disease Overview
    • Epidemiology Overview: Diagnosed Prevalent Cases of PKU in 2022 and 2027
    • Treatment Guidelines
    • 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
    • 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 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 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 – Mergers, Acquisitions, and Strategic Alliances in PKU by Region
    • Commercial Assessment – Key Market Players in PKU
    • Future Market Catalysts – Upcoming Market Catalysts in PKU
    • Methodology
    • Methodology – Sales Forecasts
    • Methodology – Pricing and Reimbursement
    • Methodology – Phase Transition Success Rate and Likelihood of Approval Analysis

Pricing

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