Phenylketonuria
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CLINICAL_TRIALS research · 2026-01-14Clinical Trial Update: Nutritional Impacts of Palynziq on Patients With Phenylketonuria (PKU)
A clinical trial update noting ongoing recruitment for a study evaluating the nutritional impacts of Palynziq (pegvaliase) in people with ph…
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FDA approval · 2026-01-08FDA Approved: SAPROPTERIN DIHYDROCHLORIDE — Original Application (Micro Labs Limited)
FDA approves an original-application Abbreviated New Drug Application for sapropterin dihydrochloride by Micro Labs Limited. This approval m…
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CLINICAL_TRIALS research · 2026-01-06Clinical Trial Update: To Evaluate the Safety and Efficacy of GS1168 Injection in Adult Phenylketonuria [EARLY_Phase 1]
This clinical trial update announces an early Phase 1 study to evaluate the safety and potential efficacy of GS1168 Injection in adults with…
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CLINICAL_TRIALS trial_result · 2025-12-29Clinical Trial Update: Preliminary Study of Brain Effects of Palynziq-Related Changes in Phenylalanine in Individuals With PKU
This completed preliminary clinical trial evaluated how Palynziq-related changes in phenylalanine impact brain function in individuals with …
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CLINICAL_TRIALS research · 2025-12-23Clinical Trial Update: Development of Novel Psychological Assessment Tools and Anxiety Intervention for Phenylketonuria [NA]
A University of Missouri-Columbia-recruited study aims to develop novel psychological assessment tools and an anxiety intervention for indiv…
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CLINICAL_TRIALS trial_result · 2025-12-22Clinical Trial Update: Study of Low-grade Systemic Inflammation in Adult Patients With Phenylketonuria [NA]
This clinical trial update indicates that the study examining low-grade systemic inflammation in adult phenylketonuria patients has complete…
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CLINICAL_TRIALS research · 2025-12-19Clinical Trial Update: GMP Powdered Substitutes in PKU and TYR [NA]
A Nutricia UK Ltd-sponsored clinical trial evaluating GMP powdered substitutes for phenylketonuria and tyrosinemia is currently recruiting.
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EMA approval · 2025-12-02EMA Authorised: Upstaza (Eladocagene exuparvovec) — PTC Therapeutics International Limited
The European Medicines Agency has authorised Upstaza (eladocagene exuparvovec) for the treatment of phenylketonuria, an inborn error of amin…
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CLINICAL_TRIALS research · 2025-12-02Clinical Trial Update: AAV Gene Therapy Clinical Study in Adult Classic PKU (PHEdom) [Phase 1, Phase 2]
An adeno-associated virus (AAV) gene therapy study for adults with classic phenylketonuria (PKU) is in Phase 1/2 and currently recruiting.
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CLINICAL_TRIALS research · 2025-12-01Clinical Trial Update: Evaluation of BH4 Responsiveness in Our PKU Patients [Phase 1]
Phase 1 clinical trial evaluating BH4 (tetrahydrobiopterin) responsiveness in phenylketonuria patients; the study is currently active but no…
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CLINICAL_TRIALS trial_result · 2025-11-26Clinical Trial Update: PKU EASY Microtabs Plus - Acceptability and Tolerance [NA]
The completed trial update reports on the acceptability and tolerability of PKU EASY Microtabs Plus for phenylketonuria. No additional effic…
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FDA approval · 2025-11-20FDA Approved: PALYNZIQ — Supplemental Application (BioMarin Pharmaceutical Inc.)
The FDA approved a supplemental application for Palynziq (pegvaliase-pqpz) from BioMarin Pharmaceutical.
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CLINICAL_TRIALS research · 2025-09-23Clinical Trial Update: A Phase 1/Phase 2 Open-label Study to Evaluate the Safety, Tolerability, and Efficacy of a Single Intravenous Administration of SAR444836 in Adult Participants With Phenylketonuria [Phase 1, Phase 2]
This clinical trial update describes a Phase 1/Phase 2 open-label study to evaluate the safety, tolerability, and efficacy of a single intra…
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CLINICAL_TRIALS trial_result · 2025-09-15Clinical Trial Update: PheCheck Feasibility Study
Lumos Diagnostics reports that the PheCheck feasibility study for phenylketonuria has completed. The update outlines preliminary findings an…
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CLINICAL_TRIALS trial_result · 2025-09-12Clinical Trial Update: Study to Evaluate the Safety and Efficacy of Pegvaliase in Adolescents (Ages 12-17) With Phenylketonuria [Phase 3]
Phase 3 trial evaluating pegvaliase for phenylketonuria in adolescents (ages 12-17) is currently active but not recruiting.
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FDA approval · 2025-09-03FDA Approved: SAPROPTERIN DIHYDROCHLORIDE — Original Application (TRUPHARMA, LLC)
FDA approved the original application for sapropterin dihydrochloride from Trupharma, LLC, for the treatment of phenylketonuria by reducing …
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CLINICAL_TRIALS research · 2025-07-31Clinical Trial Update: First-in-Human, Multiple Part Clinical Study of JNT-517 in Healthy Participants and in Participants With Phenylketonuria [Phase 1, Phase 2]
This clinical trial update reports a first-in-human, multi-part Phase 1/2 study of JNT-517 in healthy participants and in individuals with p…
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FDA approval · 2025-06-11FDA Approved: SAPROPTERIN DIHYDROCHLORIDE — Original Application (Hikma Pharmaceuticals USA Inc.)
FDA approved Hikma Pharmaceuticals USA Inc.'s sapropterin dihydrochloride via an original application, adding a new approved product for man…
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FDA approval · 2025-06-09FDA Approved: SAPROPTERIN DIHYDROCHLORIDE — Original Application (Aurobindo Pharma Limited)
FDA approved an original ANDA for sapropterin dihydrochloride from Aurobindo Pharma Limited.
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CLINICAL_TRIALS trial_result · 2025-06-08Clinical Trial Update: Evaluation of Phe Fluctuation in PKU Pts Treated With PKU GOLIKE Versus Standard Amino Acid Protein Substitute. [NA]
This completed clinical trial compared phenylalanine fluctuations in phenylketonuria patients treated with PKU GOLIKE versus a standard amin…
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CLINICAL_TRIALS research · 2025-05-15Clinical Trial Update: Phe for Me? The Effects of L-Phe on PKU Carriers and Non-carriers [NA]
This University of Guelph study investigates the effects of the amino acid L-phenylalanine on individuals who are carriers of phenylketonuri…
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CLINICAL_TRIALS research · 2025-05-13Clinical Trial Update: Brain Aging in Phenylketonuria
A recruiting clinical trial is investigating brain aging in phenylketonuria (PKU). Sponsored by Insel Gruppe AG and University Hospital Bern…
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FDA approval · 2025-04-29FDA Approved: ZELVYSIA — Original Application (Aucta Pharmaceuticals, Inc.)
The FDA approved Zelvysia (sapropterin dihydrochloride) via an original Abbreviated New Drug Application from Aucta Pharmaceuticals, Inc. fo…
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CLINICAL_TRIALS research · 2025-04-23Clinical Trial Update: A Self-test Home-use Blood Phenylalanine Monitoring System Under the Brand Name Egoo Phe System Has Been Developed for Measurement of Phenylalanine (Phe) in Individuals Diagnosed With Phenylketonuria (PKU): The Study Purpose is to Evaluate Accuracy and Usability
A clinical trial is evaluating the accuracy and usability of the home-use Egoo Phe System for measuring phenylalanine levels in people with …
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CLINICAL_TRIALS research · 2025-04-13Clinical Trial Update: Metabolic Control and Patient Well-being in Phenylketonuria: do Guidelines Make a Difference?
A recruiting clinical trial at University Children's Hospital, Zurich is investigating whether guidelines improve metabolic control and pati…