Phenylketonuria
📧 Sign up to get email alerts when something new happens for Phenylketonuria.
Sign up freeTop drugs studied for this disease
-
CLINICAL_TRIALS trial_result · 2023-04-24Clinical Trial Update: Kuvan®'s Effect on the Cognition of Children With Phenylketonuria [Phase 4]
Phase 4 trial assessing Kuvan® (tetrahydrobiopterin) effect on cognition in children with phenylketonuria has completed.
-
CLINICAL_TRIALS trial_result · 2023-03-07Clinical Trial Update: Phenylalanine and Its Impact on Cognition [Phase 4]
Phase 4 clinical trial update examining phenylalanine and its impact on cognition in phenylketonuria.
-
CLINICAL_TRIALS trial_result · 2022-10-17Clinical Trial Update: Efficacy and Safety of SYNB1618 and SYNB1934 in Adult Patients With Phenylketonuria [Phase 2]
Phase 2 trial update on the efficacy and safety of SYNB1618 and SYNB1934 in adults with phenylketonuria has been completed.
-
FDA approval · 2022-09-26FDA Approved: JAVYGTOR — Supplemental Application (Dr. Reddys Laboratories Inc)
The FDA approved a supplemental application for JAVYGTOR (sapropterin dihydrochloride) submitted by Dr. Reddys Laboratories Inc.
-
FDA approval · 2022-08-23FDA Approved: SAPROPTERIN DIHYDROCHLORIDE — Original Application (Camber Pharmaceuticals, Inc.)
FDA approved an original application for sapropterin dihydrochloride from Camber Pharmaceuticals, a generic version of the product. The drug…
- Want full summaries, importance scores, and drug timelines? Upgrade to Plus or Pro.
-
FDA approval · 2022-08-18FDA Approved: SAPROPTERIN DIHYDROCHLORIDE — Original Application (Camber Pharmaceuticals, Inc.)
FDA approved Camber Pharmaceuticals' original application for sapropterin dihydrochloride, a generic version of the PKU therapy. This action…
-
FDA approval · 2022-05-26FDA Approved: JAVYGTOR — Supplemental Application (Dr. Reddys Laboratories Inc)
FDA approved a supplemental application for JAVYGTOR (sapropterin dihydrochloride) from Dr. Reddys Laboratories Inc.
-
FDA approval · 2022-05-17FDA Approved: JAVYGTOR — Supplemental Application (Dr. Reddys Laboratories Inc)
FDA approved a supplemental application for JAVYGTOR (sapropterin dihydrochloride) from Dr. Reddy's Laboratories, updating/expanding its lab…
-
FDA approval · 2022-05-13FDA Approved: JAVYGTOR — Original Application (Dr. Reddys Laboratories Inc)
FDA approved the original application (ANDA) for JAVYGTOR, a generic sapropterin dihydrochloride, from Dr. Reddys Laboratories Inc.; it is i…
-
CLINICAL_TRIALS trial_result · 2022-04-27Clinical Trial Update: The Effectiveness of Kuvan in Amish PKU Patients [Phase 4]
Phase 4 trial reporting on the effectiveness of Kuvan in Amish patients with phenylketonuria has been completed with results posted.
-
CLINICAL_TRIALS trial_result · 2022-02-17Clinical Trial Update: Safety and Efficacy of Self Administered Injections of Pegvaliase (>40mg/Day Dose) in Adults With PKU [Phase 3]
Phase 3 trial evaluating safety and efficacy of self-administered pegvaliase (>40 mg/day) in adults with phenylketonuria (PKU) has completed…
-
CLINICAL_TRIALS trial_result · 2021-10-12Clinical Trial Update: Long-Term Extension of Previous rAvPAL-PEG Protocols in Subjects With PKU (PAL-003) [Phase 2]
Phase 2 long-term extension trial update for rAvPAL-PEG in phenylketonuria (PKU) completed.
-
FDA approval · 2021-07-26FDA Approved: SAPROPTERIN DIHYDROCHLORIDE — Supplemental Application (ENDO USA, Inc.)
The FDA approved a supplemental application for sapropterin dihydrochloride, updating its approval via ENDO USA, Inc. for use in phenylketon…
-
FDA approval · 2021-06-15FDA Approved: JAVYGTOR — Original Application (Dr. Reddys Laboratories Inc)
FDA approved JAVYGTOR (sapropterin dihydrochloride) from Dr. Reddy's Laboratories as an original ANDA, providing a generic option for phenyl…
-
CLINICAL_TRIALS trial_result · 2021-05-13Clinical Trial Update: Safety and Tolerability of SYNB1618 in Healthy Adult Volunteers and Adult Subjects With Phenylketonuria [Phase 1, Phase 2]
Phase 1/2 clinical trial evaluating safety and tolerability of SYNB1618 in healthy adults and adults with phenylketonuria has completed.
-
FDA approval · 2021-03-30FDA Approved: JAVYGTOR — Original Application (Dr. Reddys Laboratories Inc)
FDA approved JAVYGTOR (sapropterin dihydrochloride) from Dr. Reddy's Laboratories. It is a tetrahydrobiopterin (BH4) analogue used to treat …
-
CLINICAL_TRIALS trial_result · 2020-12-22Clinical Trial Update: Effect of Kuvan on Neurocognitive Function, Blood Phenylalanine Level, Safety, and Pharmacokinetics in Children With PKU [Phase 3]
A Phase 3 trial evaluating Kuvan (tetrahydrobiopterin) effects on neurocognitive function, phenylalanine levels, safety, and pharmacokinetic…
-
FDA approval · 2020-11-20FDA Approved: PALYNZIQ — Supplemental Application (BioMarin Pharmaceutical Inc.)
The FDA approved BioMarin's supplemental application for Palynziq (pegvaliase-pqpz), updating the existing approval for phenylketonuria.
-
FDA approval · 2020-10-06FDA Approved: PALYNZIQ — Supplemental Application (BioMarin Pharmaceutical Inc.)
The FDA approved a supplemental application for PALYNZIQ (pegvaliase-pqpz) from BioMarin Pharmaceutical, resulting in changes to the drug's …
-
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…
-
FDA approval · 2020-09-09FDA Approved: SAPROPTERIN DIHYDROCHLORIDE — Supplemental Application (ENDO USA, Inc.)
FDA approved a supplemental application for sapropterin dihydrochloride submitted by ENDO USA, Inc., marking an additional approval for this…
-
FDA approval · 2020-09-09FDA Approved: SAPROPTERIN DIHYDROCHLORIDE — Supplemental Application (ENDO USA, Inc.)
FDA approved a supplemental application for sapropterin dihydrochloride by ENDO USA, Inc., updating regulatory authorization for this produc…
-
CLINICAL_TRIALS trial_result · 2020-06-04Clinical Trial Update: A Phase 3 Substudy to Evaluate Executive Function in Adults With PKU Who Are Participating in the Phase 3 Study, 165-302
Phase 3 substudy assessing executive function in adults with phenylketonuria participating in the main Phase 3 trial has completed; results …
-
FDA approval · 2020-03-16FDA Approved: KUVAN — Supplemental Application (BioMarin Pharmaceutical Inc.)
The FDA approved a supplemental application for Kuvan (sapropterin dihydrochloride) by BioMarin Pharmaceutical, updating the product's label…
-
FDA approval · 2020-03-16FDA Approved: KUVAN — Supplemental Application (BioMarin Pharmaceutical Inc.)
The FDA approved a supplemental application for Kuvan (sapropterin dihydrochloride) from BioMarin Pharmaceutical Inc.