Phenylketonuria Pipeline Outlook 2025: Clinical Trial Studies, EMA, PDMA, FDA Approvals, MOA, ROA, NDA, IND, and Companies

elveInsight's, "Phenylketonuria Pipeline Insight 2025" report provides comprehensive insights about 20+ companies and 25+ pipeline drugs in Phenylketonuria pipeline landscape. It covers the Phenylketonuria pipeline drug profiles, including clinical and nonclinical stage products. It also covers the Phenylketonuria pipeline therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

Discover the latest drugs and treatment options in the Phenylketonuria Pipeline. Dive into DelveInsight's comprehensive report today! @ Phenylketonuria Pipeline Outlook [https://www.delveinsight.com/sample-request/phenylketonuria-pku-pipeline-insight?utm_source=abnewswire&utm_medium=pressrelease&utm_campaign=ypr]

Key Takeaways from the Phenylketonuria Pipeline Report

* On 22 August 2025, BioMarin Pharmaceutical conducted a Phase 3 study is designed to evaluate the safety and efficacy of pegvaliase administered daily to adolescents (ages 12 to 17 years old (US), inclusive, and 12 to 15 years old (EU), inclusive 12-17) with phenylketonuria (PKU). Participants will be randomized in a 2:1 ratio to the active (pegvaliase) and control (diet-only) treatment arms, respectively, with 36 participants receiving pegvaliase and 18 participants managing their PKU with diet alone.
* On 21 August 2025, Sanofi organized a study is constituted of 2 separate parts: a dose escalation part, and a dose expansion part where subsequent participants will be administered a safe and effective dose level identified during the dose escalation part. In both study parts, clinical and laboratory assessments will be collected to: a) assess the incidence of adverse events, and b) evaluate the effect of SAR444836 on reductions in blood Phe levels and maintenance of these Phe levels after elimination of a Phe restricted diet.
* On 19 August 2025, Otsuka Pharmaceutical Development & Commercialization, Inc. announced a Phase 2 study is to assess the safety, tolerability, and pharmacokinetics (PK) of oral JNT-517 in adolescents (12 to less than 18 years of age) with PKU. Participants will receive either JNT-517 or placebo and will be blinded to their treatment assignment. Participants will have a 4 in 5 (or 80%) chance of receiving JNT-517. The study will last for up to 63 days including a Screening period, Treatment period and Follow-up period for safety.
* DelveInsight's Phenylketonuria pipeline report depicts a robust space with 20+ active players working to develop 25+ pipeline therapies for Phenylketonuria treatment.
* The leading Phenylketonuria Companies such as PTC Therapeutics, BioMarin Pharmaceutical, Otsuka Pharmaceutical, Relief Therapeutics Holding, BioMarin Pharmaceutical, Tessera Therapeutics and others.
* Promising Phenylketonuria Pipeline Therapies such as Sepiapterin, PTC923, SYNB1618, SYNB1934, SAR444836, Kuvan Registered , Sapropterin dihydrochloride and others.

Stay ahead with the most recent pipeline outlook for Phenylketonuria. Get insights into clinical trials, emerging therapies, and leading companies with DelveInsight @ Phenylketonuria Treatment Drugs [https://www.delveinsight.com/sample-request/phenylketonuria-pku-pipeline-insight?utm_source=abnewswire&utm_medium=pressrelease&utm_campaign=ypr]

Phenylketonuria Emerging Drugs

* Sepiapterin: PTC Therapeutics

Sepiapterin (formerly PTC923), an oral formulation of synthetic sepiapterin, has a dual mechanism of action to increase activity of the phenylalanine hydroxylase (PAH) enzyme. First, sepiapterin is a precursor compound that is rapidly absorbed and converted intracellularly to tetrahydrobiopterin (BH4), a critical cofactor of PAH. Sepiapterin also has an independent chaperone effect, protecting against PAH misfolding to enhance the enzyme function. Through this dual mechanism of action, sepiapterin effectively reduces blood phenylalanine (Phe) levels and has the potential to treat the broad range of PKU patients. Currently the drug is in been registered for Phenylketonuria (PKU).

* JNT-517: Otsuka Pharmaceutical

JNT-517 is a selective small molecule inhibitor of the Phe transporter SLC6A19 that has the potential to be a first-in-class oral therapy used to treat any person with PKU, regardless of age or genotype. JNT-517 acts at a novel, cryptic allosteric site to block kidney reabsorption of Phe and offers a promising new approach to reduce blood. . Currently the drug is in Phase III for Phenylketonuria (PKU).

* RLF OD032: Relief Therapeutics Holding

RLF-OD032, an innovative and highly concentrated liquid formulation of sapropterin dihydrochloride, is designed to lower blood phenylalanine in adult and pediatric PKU patients. It offers a more patient-friendly solution by significantly reducing the volume of medication required compared to current formulations. This advancement aims to enhance compliance, particularly among pediatric patients, who often struggle with the high volumes associated with existing sapropterin treatments. If approved, RLF-OD032 would be the first and only portable, ready-to-use liquid formulation of sapropterin dihydrochloride. Currently the drug is in Phase I Stage of its development for the treatment of Phenylketonuria (PKU).

The Phenylketonuria Pipeline Report Provides Insights into

* The report provides detailed insights about companies that are developing therapies for the treatment of Phenylketonuria with aggregate therapies developed by each company for the same.
* It accesses the Different therapeutic candidates segmented into early-stage, mid-stage, and late-stage of development for Phenylketonuria Treatment.
* Phenylketonuria Companies are involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.
* Phenylketonuria 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

Explore groundbreaking therapies and clinical trials in the Phenylketonuria Pipeline. Access DelveInsight's detailed report now! @ New Phenylketonuria Drugs [https://www.delveinsight.com/sample-request/phenylketonuria-pku-pipeline-insight?utm_source=abnewswire&utm_medium=pressrelease&utm_campaign=ypr]

Phenylketonuria Companies

PTC Therapeutics, BioMarin Pharmaceutical, Otsuka Pharmaceutical, Relief Therapeutics Holding, BioMarin Pharmaceutical, Tessera Therapeutics and others.

Phenylketonuria Pipeline Report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as

* Intravenous
* Subcutaneous
* Oral
* Intramuscular

Phenylketonuria Products have been categorized under various Molecule types such as

* Monoclonal antibody
* Small molecule
* Peptide

Unveil the future of Phenylketonuria Treatment. Learn about new drugs, pipeline developments, and key companies with DelveInsight's expert analysis @ Phenylketonuria Market Drivers and Barriers [https://www.delveinsight.com/sample-request/phenylketonuria-pku-pipeline-insight?utm_source=abnewswire&utm_medium=pressrelease&utm_campaign=ypr]

Scope of the Phenylketonuria Pipeline Report

* Coverage- Global
* Phenylketonuria Companies- PTC Therapeutics, BioMarin Pharmaceutical, Otsuka Pharmaceutical, Relief Therapeutics Holding, BioMarin Pharmaceutical, Tessera Therapeutics and others.
* Phenylketonuria Pipeline Therapies- Sepiapterin, PTC923, SYNB1618, SYNB1934, SAR444836, Kuvan Registered , Sapropterin dihydrochloride and others.
* 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

Get the latest on Phenylketonuria Therapies and clinical trials. Download DelveInsight's in-depth pipeline report today! @ Phenylketonuria Companies, Key Products and Unmet Needs [https://www.delveinsight.com/sample-request/phenylketonuria-pku-pipeline-insight?utm_source=abnewswire&utm_medium=pressrelease&utm_campaign=ypr]

Table of Contents

* Introduction
* Executive Summary
* Phenylketonuria: Overview
* Pipeline Therapeutics
* Therapeutic Assessment
* Phenylketonuria- DelveInsight's Analytical Perspective
* Late Stage Products (Phase III)
* JNT-517: Otsuka Pharmaceutical
* Drug profiles in the detailed report.....
* Mid Stage Products (Phase II)
* Drug Name: Company Name
* Drug profiles in the detailed report.....
* Early Stage Products (Phase I)
* RLF OD032: Relief Therapeutics Holding
* Drug profiles in the detailed report.....
* Preclinical and Discovery Stage Products
* Drug name : Company name
* Drug profiles in the detailed report.....
* Inactive Products
* Phenylketonuria Key Companies
* Phenylketonuria Key Products
* Phenylketonuria- Unmet Needs
* Phenylketonuria- Market Drivers and Barriers
* Phenylketonuria- Future Perspectives and Conclusion
* Phenylketonuria Analyst Views
* Phenylketonuria Key Companies
* Appendix

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.

Media Contact
Company Name: DelveInsight Business Research LLP
Contact Person: Yash Bhardwaj
Email:Send Email [https://www.abnewswire.com/email_contact_us.php?pr=phenylketonuria-pipeline-outlook-2025-clinical-trial-studies-ema-pdma-fda-approvals-moa-roa-nda-ind-and-companies]
Phone: 09650213330
Address:304 S. Jones Blvd #2432
City: Las Vegas
State: NV
Country: United States
Website: https://www.delveinsight.com/report-store/phenylketonuria-pku-pipeline-insight

Legal Disclaimer: Information contained on this page is provided by an independent third-party content provider. ABNewswire makes no warranties or responsibility or liability for the accuracy, content, images, videos, licenses, completeness, legality, or reliability of the information contained in this article. If you are affiliated with this article or have any complaints or copyright issues related to this article and would like it to be removed, please contact retract@swscontact.com



This release was published on openPR.