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Phenylketonuria (PKU) Pipeline Analysis, 2022 | Insights into the Latest FDA, EMA, and PMDA Approvals, Clinical and Non-Clinical Trials, Emerging Therapies, Treatment Algorithms, and Key Players

Phenylketonuria (PKU) Pipeline Analysis, 2022 | Insights into the Latest FDA, EMA, and PMDA Approvals, Clinical and Non-Clinical Trials, Emerging Therapies, Treatment Algorithms, and Key Players

December 07
04:40 2022
Phenylketonuria (PKU) Pipeline Analysis, 2022 | Insights into the Latest FDA, EMA, and PMDA Approvals, Clinical and Non-Clinical Trials, Emerging Therapies, Treatment Algorithms, and Key Players
DelveInsight Business Research LLP
As per DelveInsight’s assessment, globally, about 10+ key pharma and biotech companies are working on 15+ pipeline drugs in the Phenylketonuria (PKU) therapeutics landscape based on different Routes of Administration (RoA), Mechanism of Action (MoA) and molecule types. Several of the therapies are in the advanced stages of clinical development and are expected to launch in the coming years.

SYNB-1618 is the most anticipated emerging therapy, awaiting launch. Apart from this, other products in the Phenylketonuria (PKU) therapeutic pipeline segment include CNSA-001, BMN 307, and one gene therapy, HMI-102. Some of these products have also received Fast track and Orphan Designations. Key players, such as Synlogic, Censa Pharmaceuticals, Codexis, Homology Medicines, etc., are developing therapies for the treatment of patients with Phenylketonuria.

As per DelveInsight, the increasing awareness, growing research in the field of genomics and bioinformatics, changing lifestyles, advancements in the healthcare industry, and rising per capita income in emerging markets are expected to drive the Phenylketonuria diagnosis and treatment market in the coming year’s. Moreover, the rising mergers and acquisitions and collaboration activities among the major market participants are also estimated to drive the Phenylketonuria therapeutics market growth.

 “Phenylketonuria (PKU) Pipeline Insight, 2022” report by DelveInsight provides a comprehensive analysis of the ongoing clinical development activities and growth prospects across the Phenylketonuria Therapeutics Market. 

The report provides a detailed description of the drug, including the mechanism of action, type, stage, route of administration, molecule type, clinical studies, inactive pipeline products, and NDA approvals (if any). 

The report also covers commercial and clinical activities of the pipeline products from the pre-clinical developmental phase to the marketed stage. Additionally, it presents the ongoing developments in the therapeutics segment, including collaborations, licensing, mergers & acquisitions (M&A), funding, designations, and other product-related details.

Phenylketonuria (PKU) Pipeline Analysis

The report provides insights into: 

  • The report provides detailed insights into the key companies that are developing therapies in the Phenylketonuria Market.

  • The report also evaluates different therapeutic candidates segmented into early-stage, mid-stage, and late-stage of development for Phenylketonuria treatment.

  • It analyzes the key companies involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.

  • It navigates the emerging drugs under development based on the stage of development, route of administration, target receptor, monotherapy or combination therapy, a different mechanism of action, and molecular type. 

  • Detailed analysis of collaborations (company-company collaborations and company-academia collaborations), licensing agreement, and financing details for future advancement of the Phenylketonuria market.

The report is built using data and information traced from the researcher’s proprietary databases, company/university websites, clinical trial registries, conferences, SEC filings, investor presentations, and featured press releases from company/university websites and industry-specific third-party sources, etc. 

The Report Covers the Emerging Pipeline Products Under Different Phases of Clinical Development Like – 

  • Late-stage products (Phase III)

  • Mid-stage products (Phase II)

  • Early-stage products (Phase I) 

  • Pre-clinical and Discovery stage candidates

  • Discontinued & Inactive candidates

Phenylketonuria (PKU) – Mechanism of Action (MOA)

  • Mixed-function oxygenase modulator

  • Gastrointestinal microbiome modulator

  • Phenylalanine ammonia-lyase replacement

  • Gene transference

  • SLC6A19 protein inhibitor

  • Phenylalanine hydroxylase stimulant

  • Phenylalanine hydroxylase replacement

Request for Sample PDF to Understand More About the Treatment Outlook and Future Perspectives  @

Phenylketonuria (PKU) Therapeutics Analysis 

The management of Phenylketonuria is complex and dietary non-adherence often increases in adolescence and early adulthood, particularly due to social issues. Besides a low Phe diet, promising drugs are being developed for PKU. Oral sapropterin dihydrochloride (KUVAN), a synthetic form of BH4, could help lower Phe levels in some PKU patients. To further improve the treatment scenario, companies across the globe are diligently working toward the development of novel treatment therapies with a considerable amount of success over the years.

There are approx. 10+ key companies which are developing therapies for Phenylketonuria. PTC Therapeutics is leading the therapeutics market with its Phenylketonuria drug candidates in the most advanced stage of clinical development. 

Phenylketonuria (PKU) Therapies Covered in the Report Include:

  • Homology Medicines

  • PTC Therapeutics

  • Synlogic

  • Rubius Therapeutics

  • Agios Pharmaceuticals

  • Generation Bio

  • Jnana Therapeutics

  • Sepiapterin: PTC therapeutics

  • BioMarin Pharmaceuticals

  • Jnana Therapeutics

  • APR Applied Pharma Research

  • Censa Pharmaceuticals

And many more

Phenylketonuria (PKU) Drugs Covered in the Report Include

  • SYNB1618: Synlogic

  • HMI-102: Homology Medicines

  • HMI 103: Homology Medicines

  • SYNB1618: Synlogic

  • RTX-134: Rubius Therapeutics

  • Sepiapterin: PTC therapeutics

  • BMN-307: BioMarin Pharmaceuticals

  • JNT 517: Jnana Therapeutics

  • APR-OD031: APR Applied Pharma Research

  • CNSA-001: Censa Pharmaceuticals

And More Others

Request the Sample PDF to Get a Better Understanding of the Emerging Drugs and Key Companies @

Table of Content

1. Report Introduction

2. Executive Summary

3. Phenylketonuria Current Treatment Patterns

4. Phenylketonuria – DelveInsight’s Analytical Perspective

5. Therapeutic Assessment

6. Phenylketonuria Late Stage Products (Phase-III)

7. Phenylketonuria Mid-Stage Products (Phase-II)

8. Early Stage Products (Phase-I)

9. Pre-clinical Products and Discovery Stage Products

10. Inactive Products

11. Dormant Products

12. Phenylketonuria Discontinued Products

13. Phenylketonuria Product Profiles

14. Phenylketonuria Key Companies

15. Phenylketonuria Key Products

16. Dormant and Discontinued Products

17. Phenylketonuria Unmet Needs

18. Phenylketonuria Future Perspectives

19. Phenylketonuria Analyst Review  

20. Appendix

21. Report Methodology

*The table of contents is not exhaustive; the final content may vary. 

Download Sample PDF to Explore the Key Offerings of the Report @

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About DelveInsight
DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports Pharma companies by providing comprehensive end-to-end solutions to improve their performance.

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