Hyperphenylalaninemia
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CLINICAL_TRIALS research · 2026-03-20Clinical Trial Update: Phenylalanine-free Diet for Patients With Secondary Hyperphenylalaninemia in ICU [NA]
This clinical trial record indicates a phenylalanine-free diet is being studied for patients with secondary hyperphenylalaninemia in the ICU…
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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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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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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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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A clinical trial investigating a low-calorie PKU drink sponsored by Nutricia UK Ltd has been withdrawn.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 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 · 2023-11-14Clinical Trial Update: A Study of PTC923 (CNSA-001) in Primary Tetrahydrobiopterin (BH4) Deficient Participants With Hyperphenylalaninemia [Phase 1, Phase 2]
Phase 1/2 trial of PTC923 (CNSA-001) in participants with primary BH4 deficiency and hyperphenylalaninemia has completed with results posted…
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CLINICAL_TRIALS trial_result · 2020-09-25Clinical Trial Update: Safety and Efficacy Study of Phenoptin in Subjects With Hyperphenylalaninemia Due to BH4 Deficiency [Phase 2]
Phase 2 safety and efficacy study of Phenoptin in subjects with hyperphenylalaninemia due to tetrahydrobiopterin deficiency has completed wi…
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FDA approval · 2020-09-09FDA Approved: SAPROPTERIN DIHYDROCHLORIDE — Supplemental Application (ENDO USA, Inc.)
The FDA approved a supplemental application for sapropterin dihydrochloride from Endo USA, Inc., indicating an update to the product’s label…
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FDA approval · 2019-12-13FDA Approved: KUVAN — Supplemental Application (BioMarin Pharmaceutical Inc.)
The FDA approved BioMarin's supplemental application for KUVAN (sapropterin dihydrochloride), resulting in an approved update to its labelin…
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FDA approval · 2019-08-20FDA Approved: SAPROPTERIN DIHYDROCHLORIDE — Original Application (ENDO USA, Inc.)
FDA approved an original Abbreviated New Drug Application for sapropterin dihydrochloride from ENDO USA, Inc., enabling a generic option for…
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CLINICAL_TRIALS trial_result · 2017-01-16Clinical Trial Update: Antioxidant Signature in Adult Patients With Phenylketonuria
A completed clinical trial investigated antioxidant signatures in adult patients with phenylketonuria.
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FDA approval · 2016-08-09FDA Approved: KUVAN — Supplemental Application (BioMarin Pharmaceutical Inc.)
The FDA approved a supplemental application for Kuvan (sapropterin dihydrochloride) by BioMarin Pharmaceutical Inc.
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CLINICAL_TRIALS trial_result · 2016-02-17Clinical Trial Update: Neurocognitive Outcomes in Mild Hyperphenylalaninemia (MHP)MHP Study
A completed clinical trial update evaluating neurocognitive outcomes in individuals with mild hyperphenylalaninemia/phenylketonuria; the sni…
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CLINICAL_TRIALS trial_result · 2016-02-08Clinical Trial Update: Phenylketonuria and Hyperphenylalaninemia Nutrition Study
This entry reports the completion of a nutrition-focused clinical trial studying phenylketonuria and hyperphenylalaninemia. The provided sni…
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CLINICAL_TRIALS trial_result · 2015-12-11Clinical Trial Update: Biological Variation of Phenylalanine in Patients With Hyperphenylalaninemia
A completed clinical trial examining the biological variation of phenylalanine levels in patients with hyperphenylalaninemia/phenylketonuria…
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CLINICAL_TRIALS trial_result · 2015-07-07Clinical Trial Update: Bone Mineral Density in Adults With Hyperphenylalaninemia on Kuvan Therapy [NA]
This clinical trial update reports bone mineral density changes in adults with hyperphenylalaninemia treated with Kuvan. The study (NCT01541…
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CLINICAL_TRIALS trial_result · 2015-06-12Clinical Trial Update: Bone Mineral Density in Adults With Hyperphenylalaninemia
This clinical trial update assesses bone mineral density in adults with hyperphenylalaninemia (phenylketonuria). The update summarizes the b…
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FDA approval · 2013-11-22FDA Approved: KUVAN — Supplemental Application (BioMarin Pharmaceutical Inc.)
FDA approved a supplemental application for KUVAN (sapropterin dihydrochloride) submitted by BioMarin, resulting in an updated labeling for …