Phenylketonuria
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Sign up freeTop drugs studied for this disease
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CLINICAL_TRIALS trial_result · 2020-01-13Clinical Trial Update: BH4 Responsiveness in PAH Deficiency PKU Patients [Phase 4]
Phase 4 clinical trial update on BH4 responsiveness in phenylketonuria patients with PAH deficiency.
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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 · 2019-12-13FDA Approved: KUVAN — Supplemental Application (BioMarin Pharmaceutical Inc.)
FDA approved a supplemental application for Kuvan (sapropterin dihydrochloride) from BioMarin Pharmaceutical, updating its labeling.
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CLINICAL_TRIALS trial_result · 2019-10-08Clinical Trial Update: Kuvan Therapy in Phenylketonuria (PKU): The Effect of Blood Phenylalanine Concentration on Kuvan Response [Phase 2]
Phase 2 trial update evaluating how blood phenylalanine levels influence Kuvan response in phenylketonuria.
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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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FDA approval · 2019-08-20FDA Approved: SAPROPTERIN DIHYDROCHLORIDE — Original Application (ENDO USA, Inc.)
FDA approves a generic sapropterin dihydrochloride product under ANDA 210027 (Original Application) by ENDO USA, Inc.
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CLINICAL_TRIALS trial_result · 2019-07-12Clinical Trial Update: Phase 3 Study to Evaluate the Efficacy & Safety of Self-Administered Injections of BMN165 by Adults With PKU [Phase 3]
Phase 3 trial evaluating self-administered injections of BMN165 for adults with phenylketonuria has completed; results were posted in 2019.
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FDA approval · 2019-05-10FDA Approved: SAPROPTERIN DIHYDROCHLORIDE — Original Application (ENDO USA, Inc.)
The FDA approved the Original Application for sapropterin dihydrochloride from ENDO USA, Inc., authorizing a generic version of the drug.
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FDA approval · 2019-05-02FDA Approved: KUVAN — Supplemental Application (BioMarin Pharmaceutical Inc.)
The FDA approved BioMarin's supplemental application for Kuvan (sapropterin dihydrochloride), expanding or updating its labeling for the pro…
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FDA approval · 2019-05-02FDA Approved: KUVAN — Supplemental Application (BioMarin Pharmaceutical Inc.)
FDA approved BioMarin's supplemental application for KUVAN (sapropterin dihydrochloride) submitted by BioMarin Pharmaceutical Inc.
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EMA approval · 2019-03-01CHMP recommends authorisation of new treatment for phenylketonuria, a rare inherited metabolic disease
The CHMP recommends authorisation of a new treatment for phenylketonuria, a rare inherited metabolic disease.
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CLINICAL_TRIALS trial_result · 2019-02-27Clinical Trial Update: A Study to Evaluate Subcutaneously Administered rAvPAL-PEG in Patients With Phenylketonuria for 24 Weeks [Phase 2]
Phase 2 clinical trial evaluating subcutaneously administered rAvPAL-PEG in phenylketonuria has completed, with results posted.
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CLINICAL_TRIALS trial_result · 2019-02-26Clinical Trial Update: Safety, Tolerability, and Efficacy Study of rAvPAL-PEG Administered Daily in Subjects With Phenylketonuria (PKU) [Phase 2]
Phase 2 trial update on rAvPAL-PEG for phenylketonuria assessing safety, tolerability, and efficacy.
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CLINICAL_TRIALS trial_result · 2019-02-26Clinical Trial Update: Dose-Finding Study to Evaluate the Safety, Efficacy, & Tolerability of Multiple Doses of rAvPAL-PEG in Subjects With PKU [Phase 2]
Phase 2 dose-finding trial evaluating safety, efficacy, and tolerability of multiple doses of rAvPAL-PEG in phenylketonuria. Results posted …
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FDA approval · 2019-02-01FDA Approved: KUVAN — Supplemental Application (BioMarin Pharmaceutical Inc.)
FDA approved a supplemental application for Kuvan (sapropterin dihydrochloride) from BioMarin Pharmaceutical Inc. to support treatment of ph…
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CLINICAL_TRIALS trial_result · 2018-09-10Clinical Trial Update: An Open-Label Phase 3 Study of BMN 165 for Adults With PKU Not Previously Treated w/ BMN 165 [Phase 3]
Open-label Phase 3 trial of BMN 165 in adults with phenylketonuria who were not previously treated with BMN 165 has completed and results we…
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CLINICAL_TRIALS trial_result · 2018-06-18Clinical Trial Update: Sapropterin in Individuals With Phenylketonuria
This clinical trial update reports that a study evaluating sapropterin for phenylketonuria has completed.
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FDA approval · 2018-05-24FDA Approved: PALYNZIQ — Original Application (BioMarin Pharmaceutical Inc.)
FDA approved PALYNZIQ (pegvaliase-pqpz) from BioMarin Pharmaceutical for the treatment of adults with phenylketonuria.
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CLINICAL_TRIALS research · 2017-10-13Clinical Trial Update: Body Composition, Energy Intake and Expenditure in People With Phenylketonuria
This is a clinical trial update examining body composition, energy intake and expenditure in people with phenylketonuria (PKU).
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CLINICAL_TRIALS trial_result · 2017-03-03Clinical Trial Update: Phase 2 Study of Glycomacropeptide Versus Amino Acid Diet for Management of Phenylketonuria [NA]
Phase 2 study comparing glycomacropeptide to amino acid diet for phenylketonuria has completed with results posted in 2017.
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CLINICAL_TRIALS trial_result · 2017-02-23Clinical Trial Update: Observational Study of Endothelial Dysfunction in Phenylketonuria
This observational trial evaluating endothelial dysfunction in individuals with phenylketonuria has completed.
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CLINICAL_TRIALS trial_result · 2017-02-01Clinical Trial Update: Safety and Tolerability Study of rAvPAL-PEG to Treat Phenylketonuria [Phase 1]
This document reports the completion of a Phase 1 safety and tolerability study of rAvPAL-PEG for phenylketonuria.
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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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CLINICAL_TRIALS trial_result · 2016-11-18Clinical Trial Update: Effects of Kuvan on Melatonin Secretion [Phase 4]
Phase 4 trial evaluating the effect of Kuvan (sapropterin dihydrochloride) on melatonin secretion in individuals with phenylketonuria has be…
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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.