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Phenylketonuria Pipeline, Emerging Drugs, Clinical Trials, and FDA Approvals 2023 (Updated)

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Phenylketonuria Pipeline, Emerging Drugs, Clinical Trials, and FDA Approvals 2023 (Updated)

September 27
22:24 2023
Phenylketonuria Pipeline, Emerging Drugs, Clinical Trials, and FDA Approvals 2023 (Updated)

DelveInsight’s, “Phenylketonuria Pipeline Insight 2023” report provides comprehensive insights about 18+ companies and 20+ pipeline drugs in the Phenylketonuria pipeline landscape. It covers the Phenylketonuria pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

 

Key Takeaways from the Phenylketonuria Pipeline Report

  • DelveInsight’s Phenylketonuria pipeline report depicts a robust space with 18+ active players working to develop 20+ pipeline therapies for Phenylketonuria treatment.
  • The leading companies working in the Phenylketonuria Market include BioMarin Pharmaceutical, Homology Medicine, Jnana Therapeutics, Nestlé Health Science, Moderna, SOM Biotech, Agios Pharmaceuticals, APR Applied Pharma Research, American Gene Technologies, Generation Bio, PTC Therapeutics, and others.
  • Promising Phenylketonuria Pipeline Therapies in the various stages of development include Kuvan®, PTC923, JNT-517 Suspension, SAR444836, SYNB1618, SYNB1934, and others.
  • September 2023: BioMarin Pharmaceutical announced a study of phase 3 clinical trials for Pegvaliase. This is a Phase 3 open-label randomized controlled study enrolling approximately 54 adolescents with PKU. The study is designed to assess the safety and efficacy of pegvaliase injections.
  • September 2023: Homology Medicines Inc. announced a study of phase 1 clinical trials for HMI-103. This is an open-label, sequential ascending dose-escalation, Phase 1 study to evaluate the safety and efficacy of a single intravenous (I.V.) administration of HMI-103, a gene editing development candidate, in adult participants aged 18 to 55 years, inclusive, with classical PKU due to PAH deficiency who have uncontrolled disease despite Phe restricted dietary management.

 

Request a sample and discover the recent advances in Phenylketonuria Treatment Drugs @ Phenylketonuria Pipeline Report

 

In the Phenylketonuria pipeline report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, Phenylketonuria collaborations, licensing, mergers and acquisition, funding, designations and other product related details.

 

Phenylketonuria Overview

Phenylketonuria as known as PKU is a metabolic disorder that can adversely affect the body’s natural homeostatic or steady state and lead to chemical imbalances and severe pathological conditions. Phenylketonuria is such an example in which the normal conversion of the dietary amino acid phenylalanine to tyrosine is blocked.

 

Find out more about Phenylketonuria Therapeutics Assessment @ Phenylketonuria Preclinical and Discovery Stage Products

 

Phenylketonuria Emerging Drugs Profile

  • Sepiapterin: PTC Therapeutics
  • SYNB1618: Synlogic
  • HMI-102: Homology Medicine

 

Phenylketonuria Pipeline Therapeutics Assessment

There are approx. 18+ key companies which are developing the Phenylketonuria therapies. The Phenylketonuria companies which have their Phenylketonuria drug candidates in the most advanced stage, i.e. phase III include, PTC Therapeutics.

 

Learn more about the emerging Phenylketonuria Pipeline Therapies @ Phenylketonuria Clinical Trials Assessment

 

Scope of the Phenylketonuria Pipeline Report

  • Coverage- Global
  • Phenylketonuria Therapeutic Assessment by Product Type: Mono, Combination, Mono/Combination
  • Phenylketonuria Therapeutic Assessment by Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III
  • Phenylketonuria Companies- BioMarin Pharmaceutical, Homology Medicine, Jnana Therapeutics, Nestlé Health Science, Moderna, SOM Biotech, Agios Pharmaceuticals, APR Applied Pharma Research, American Gene Technologies, Generation Bio, PTC Therapeutics, and others.
  • Phenylketonuria Pipeline Therapies- Kuvan®, PTC923, JNT-517 Suspension, SAR444836, SYNB1618, SYNB1934, and others.

 

Dive deep into rich insights for new drugs for Phenylketonuria treatment, Visit @ Phenylketonuria Market Drivers and Barriers, and Future Perspective

 

Table of Content

  1. Introduction
  2. Executive Summary
  3. Phenylketonuria: Overview
  4. Pipeline Therapeutics
  5. Therapeutic Assessment
  6. Phenylketonuria– DelveInsight’s Analytical Perspective
  7. Late Stage Products (Phase III)
  8. Sepiapterin: PTC Therapeutics
  9. Drug profiles in the detailed report…..
  10. Mid Stage Products (Phase II)
  11. SYNB1618: Synlogic
  12. Drug profiles in the detailed report…..
  13. Early Stage Products (Phase I)
  14. Drug name : Company name
  15. Drug profiles in the detailed report…..
  16. Preclinical and Discovery Stage Products
  17. Drug name : Company name
  18. Drug profiles in the detailed report…..
  19. Inactive Products
  20. Phenylketonuria Key Companies
  21. Phenylketonuria Key Products
  22. Phenylketonuria- Unmet Needs
  23. Phenylketonuria- Market Drivers and Barriers
  24. Phenylketonuria- Future Perspectives and Conclusion
  25. Phenylketonuria Analyst Views
  26. Phenylketonuria Key Companies
  27. Appendix

 

For further information on the Phenylketonuria pipeline therapeutics, reach out to Phenylketonuria Unmet Needs and Analyst Views

 

About Us

DelveInsight is a leading healthcare-focused market research and consulting firm that provides clients with high-quality market intelligence and analysis to support informed business decisions. With a team of experienced industry experts and a deep understanding of the life sciences and healthcare sectors, we offer customized research solutions and insights to clients across the globe. Connect with us to get high-quality, accurate, and real-time intelligence to stay ahead of the growth curve.

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