Phenylketonuria
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Sign up freeTop drugs studied for this disease
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CLINICAL_TRIALS research · 2025-04-06Clinical Trial Update: Effect of Large Neutral Amino Acids in Adults With Classical Phenylketonuria [NA]
A clinical trial is evaluating large neutral amino acids as a treatment for adults with classical Phenylketonuria and is currently recruitin…
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CLINICAL_TRIALS trial_result · 2025-04-04Clinical Trial Update: Effect of L-carnitine Supplementation on Phenylalanine and Brain-derived Neurotrophic Factor Levels in Infants and Children With Phenylketonuria [Phase 4]
This Phase 4 clinical trial update describes evaluating L-carnitine supplementation for its effect on phenylalanine and brain-derived neurot…
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CLINICAL_TRIALS research · 2025-04-04Clinical Trial Update: Early Check: Expanded Screening in Newborns
An update on an ongoing clinical trial (NCT03655223) evaluating expanded newborn screening under the Early Check program, with enrollment by…
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FDA approval · 2025-04-03FDA Approved: SAPROPTERIN DIHYDROCHLORIDE — Supplemental Application (Camber Pharmaceuticals, Inc.)
FDA approved a supplemental application for Camber Pharmaceuticals' sapropterin dihydrochloride, establishing a generic version of the treat…
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CLINICAL_TRIALS research · 2025-04-03Clinical Trial Update: Educational, Social Support, and Nutritional Interventions and Their Cumulative Effect on Pregnancy Outcomes and Quality of Life in Teen and Adult Women With Phenylketonuria [NA]
Clinical trial evaluating educational, social support, and nutritional interventions and their cumulative effect on pregnancy outcomes and q…
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CLINICAL_TRIALS trial_result · 2025-03-04Clinical Trial Update: Amino Acid Kinetics of GMP-AA in Healthy Human Volunteers [NA]
The clinical trial evaluated amino acid kinetics of GMP-AA in healthy volunteers. The study has completed.
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FDA approval · 2025-02-28FDA Approved: JAVYGTOR — Supplemental Application (Dr. Reddys Laboratories Inc)
FDA approved a supplemental application for JAVYGTOR (sapropterin dihydrochloride) from Dr. Reddys Laboratories Inc.
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CLINICAL_TRIALS withdrawal · 2025-02-26Clinical Trial Update: PKU Low Calorie Drink Study [NA]
A clinical trial investigating a low-calorie PKU drink sponsored by Nutricia UK Ltd has been withdrawn.
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FDA approval · 2025-01-24FDA Approved: JAVYGTOR — Supplemental Application (Dr. Reddys Laboratories Inc)
FDA approved a supplemental application for JAVYGTOR (sapropterin dihydrochloride) submitted by Dr. Reddys Laboratories Inc.
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FDA approval · 2025-01-24FDA Approved: SAPROPTERIN DIHYDROCHLORIDE — Supplemental Application (Camber Pharmaceuticals, Inc.)
The FDA approved a supplemental application for sapropterin dihydrochloride from Camber Pharmaceuticals, Inc., for its continued marketing a…
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CLINICAL_TRIALS trial_result · 2025-01-24Clinical Trial Update: Iron Supplements Effect on b-Phe Levels in PKU Patients During 3 Years Study [Phase 2]
A Phase 2 clinical trial evaluating the effect of iron supplements on blood phenylalanine levels in patients with phenylketonuria has comple…
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CLINICAL_TRIALS research · 2024-12-12Clinical Trial Update: AAV Gene Therapy Study for Subjects with PKU [Phase 1, Phase 2]
BioMarin's AAV-based gene therapy for phenylketonuria (PKU) is being studied in a Phase 1/Phase 2 trial and is currently active but not recr…
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CLINICAL_TRIALS trial_result · 2024-12-06Clinical Trial Update: PKU Carriers Trial (Pilot Study): Impact on Cognition, Mental Health, Blood Pressure and Metabolism [NA]
The University of Guelph is conducting a pilot study in genetic PKU carriers to evaluate effects on cognition, mental health, blood pressure…
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CLINICAL_TRIALS research · 2024-11-14Clinical Trial Update: Safety and Efficacy Study of NGGT002 in cPKU Adult Subjects [Phase 1, Phase 2]
NGGT002 is being studied in adults with phenylketonuria in Phase 1/2 trials and is currently recruiting to evaluate safety and efficacy.
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CLINICAL_TRIALS withdrawal · 2024-10-22Clinical Trial Update: A Dose-finding Study to Evaluate mRNA-3210 in Participants With Phenylketonuria [Phase 1, Phase 2]
A dose-finding study of mRNA-3210 for phenylketonuria (Phase 1/Phase 2) has been withdrawn.
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CLINICAL_TRIALS trial_result · 2024-10-09Clinical Trial Update: Determining the Most Efficient Screening Cut Off Levels for Phenylketonuria (PKU)
A completed clinical trial from Central Hospital in Nancy, France, evaluated different phenylketonuria newborn screening cutoff levels to de…
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FDA approval · 2024-08-26FDA Approved: KUVAN — Supplemental Application (BioMarin Pharmaceutical Inc.)
The FDA approved a supplemental application for Kuvan (sapropterin dihydrochloride) from BioMarin Pharmaceutical Inc.
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FDA approval · 2024-08-26FDA Approved: KUVAN — Supplemental Application (BioMarin Pharmaceutical Inc.)
FDA approved BioMarin's supplemental application for Kuvan (sapropterin dihydrochloride), updating the product under a supplemental approval…
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CLINICAL_TRIALS research · 2024-07-25Clinical Trial Update: Protein Requirements in Adults With Phenylketonuria (PKU) [NA]
This entry reports a completed clinical trial evaluating protein requirements in adults with phenylketonuria.
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EMA approval · 2024-07-12EMA Authorised: Palynziq (pegvaliase) — BioMarin International Limited
The European Medicines Agency authorised Palynziq (pegvaliase) by BioMarin International Limited for phenylketonuria, with orphan medicine d…
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CLINICAL_TRIALS trial_result · 2024-06-20Clinical Trial Update: Efficacy and Safety of SYNB1934 in Patients With PKU (SYNPHENY-3) [Phase 3]
Phase 3 trial SYNPHENY-3 evaluating SYNB1934 in phenylketonuria was terminated. Results were posted in June 2024.
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FDA label_change · 2024-03-15FDA Approved: JAVYGTOR — Supplemental Application (Dr. Reddys Laboratories Inc)
FDA approved a supplemental application for JAVYGTOR (sapropterin dihydrochloride) by Dr. Reddy's Laboratories, resulting in updated labelin…
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FDA approval · 2024-03-15FDA Approved: JAVYGTOR — Supplemental Application (Dr. Reddys Laboratories Inc)
The FDA approved a supplemental application for JAVYGTOR (sapropterin dihydrochloride) from Dr. Reddys Laboratories Inc.
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CLINICAL_TRIALS trial_result · 2024-01-10Clinical Trial Update: A Study of PTC923 in Participants With Phenylketonuria [Phase 3]
Phase 3 study of PTC923 in phenylketonuria has completed; results posted.
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FDA approval · 2023-12-09FDA Approved: PALYNZIQ — Supplemental Application (BioMarin Pharmaceutical Inc.)
The FDA approved BioMarin's supplemental Biologics License Application for PALYNZIQ (pegvaliase-pqpz). The approval covers an additional sub…