Phenylketonuria Marketed and Pipeline Drugs Assessment, Clinical Trials and Competitive Landscape
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Phenylketonuria (PKU) is caused by mutations in the gene that helps make an enzyme called phenylalanine hydroxylase (PAH). This enzyme is needed to convert the amino acid phenylalanine into other substances the body needs. When this gene, known as the PAH gene, is defective, the body cannot break down phenylalanine. Without treatment, PKU can damage the brain and nervous system, which can lead to learning disabilities.
The Phenylketonuria marketed and pipeline drugs market research report includes an assessment of the disease epidemiology, leading marketed products with sales forecast, pricing and reimbursement, late-to-mid-stage pipeline products with phase transition success rate (PTSR), and the likelihood of approval (LoA) for Phenylketonuria (PKU).
Leading Marketed Drugs for Phenylketonuria
The leading marketed drugs for Phenylketonuria are sapropterin dihydrochloride and pegvaliase.
Phenylketonuria Marketed Drugs Segmentation by Mechanism of Action
The key mechanisms of action for Phenylketonuria marketed drugs are enzyme activator and enzyme replacement.
Phenylketonuria Marketed Drugs Analysis, by Mechanism of Action
For more MoA insights into the Phenylketonuria marketed drugs, download a free report sample
Phenylketonuria Marketed Drugs Segmentation by Routes of Administration
The key routes of administration for Phenylketonuria marketed drugs are oral and injection.
Phenylketonuria Marketed Drugs Analysis, by Routes of Administration
For more routes of administration insights into the Phenylketonuria marketed drugs, download a free report sample
Phenylketonuria Marketed Drugs Segmentation by Molecule Type
The key molecule types for Phenylketonuria marketed drugs are small molecule and biologic.
Phenylketonuria Marketed Drugs Analysis, by Molecule type
For more molecule type insights into the Phenylketonuria marketed drugs, download a free report sample
Leading Pipeline Drugs for Phenylketonuria
Some of the leading pipeline drugs for Phenylketonuria are pegvaliase, sepiapterin, bevufenogene nofeparvovec, SYNB-1618, and SYNB-1934.
Phenylketonuria Pipeline Drugs Segmentation by Mechanism of Action
The key mechanisms of action for Phenylketonuria pipeline drugs are enzyme activator, enzyme replacement, ligand replacement, and transporter inhibitor.
Phenylketonuria Pipeline Drugs Analysis, by Mechanisms of Action
For more MoA insights into the Phenylketonuria pipeline drugs, download a free report sample
Phenylketonuria Pipeline Drugs Segmentation by Routes of Administration
The key routes of administration for Phenylketonuria pipeline drugs are oral and injection.
Phenylketonuria Pipeline Drugs Analysis, by Routes of Administration
For more routes of administration insights into the Phenylketonuria pipeline drugs, download a free report sample
Phenylketonuria Pipeline Drugs Segmentation by Molecule Type
The key molecule types for Phenylketonuria pipeline drugs are biologic, small molecule, oligonucleotide, and polymer.
Phenylketonuria Pipeline Drugs Analysis, by Molecule Types
For more molecule type insights into the Phenylketonuria pipeline drugs, download a free report sample
Top Sponsors in the Phenylketonuria Marketed and Pipeline Drugs Market
Some of the top sponsors in the Phenylketonuria marketed and pipeline drugs market are BioMarin Pharmaceutical Inc, Vitaflo International Ltd, Synlogic Inc, PTC Therapeutics Inc, Nutricia Ltd, Codexis Inc, Nestle Health Science, and Homology Medicines Inc.
Phenylketonuria Marketed Drugs Report Overview
Key Mechanisms of Action | Enzyme Activator and Enzyme Replacement |
Key Routes of Administration | Oral and Injection |
Key Molecule Types | Small Molecule and Biologic |
Phenylketonuria Pipeline Drugs Report Overview
Key Mechanisms of Action | Enzyme Activator, Enzyme Replacement, Ligand Replacement, and Transporter Inhibitor |
Key Routes of Administration | Oral and Injection |
Key Molecule Types | Biologic, Small Molecule, Oligonucleotide, and Polymer |
Top Sponsors (Marketed and Pipeline Drugs) | BioMarin Pharmaceutical Inc, Vitaflo International Ltd, Synlogic Inc, PTC Therapeutics Inc, Nutricia Ltd, Codexis Inc, Nestle Health Science, and Homology Medicines Inc.
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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 shaping and driving the Phenylketonuria market.
- Drive revenues by understanding the key trends, innovative products and technologies, market segments, and companies likely to impact the 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
Frequently asked questions
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What are the key mechanisms of action for Phenylketonuria marketed drugs?
The key mechanisms of action for Phenylketonuria marketed drugs are enzyme activator and enzyme replacement.
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What are the key routes of administration for Phenylketonuria marketed drugs?
The key routes of administration for Phenylketonuria marketed drugs are oral and injection.
-
What are the key molecule types for Phenylketonuria marketed drugs?
The key molecule types for Phenylketonuria marketed drugs are small molecule and biologic.
-
What are the key mechanisms of action for Phenylketonuria pipeline drugs?
The key mechanisms in the Phenylketonuria pipeline drugs market are enzyme activator, enzyme replacement, ligand replacement, and transporter inhibitor.
-
What are the key routes of administration for Phenylketonuria pipeline drugs?
The key routes of administration in the Phenylketonuria pipeline drugs market are oral and injection.
-
What are the key molecule types for Phenylketonuria pipeline drugs?
The key molecule types in the Phenylketonuria pipeline drugs market are biologic, small molecule, oligonucleotide, and polymer.
-
Who are the top sponsors for the Phenylketonuria marketed and pipeline drugs market?
Some of the top sponsors in the Phenylketonuria marketed and pipeline drugs market are BioMarin Pharmaceutical Inc, Vitaflo International Ltd, Synlogic Inc, PTC Therapeutics Inc, Nutricia Ltd, Codexis Inc, Nestle Health Science, and Homology Medicines Inc.
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