Trial Profile
A Pivotal bioavailability and bioequivalence Bridging Study Comparing ACER-001 with BUPHENYL in Healthy Subjects for treatment of urea-cycle-disorders
Status:
Active, no longer recruiting
Phase of Trial:
Phase III
Latest Information Update: 29 Dec 2022
Price :
$35
*
At a glance
- Drugs Sodium phenylbutyrate (Primary) ; Sodium phenylbutyrate (Primary)
- Indications Inborn urea cycle disorders; Maple syrup urine disease
- Focus Adverse reactions; Pharmacokinetics; Registrational
- Sponsors Acer Therapeutics
Most Recent Events
- 27 Dec 2022 According to an Acer Therapeutics media release, the U.S. Food and Drug Administration (FDA) has approved OLPRUVA™ (sodium phenylbutyrate) for oral suspension in the U.S. for the treatment of certain patients living with urea cycle disorders (UCDs) involving deficiencies of carbamylphosphate synthetase (CPS), ornithine transcarbamylase (OTC), or argininosuccinic acid synthetase (AS), based on data from this and other trials.
- 28 Jul 2022 According to an Acer Therapeutics media release, the U.S. Food and Drug Administration (FDA) has accepted resubmitted New Drug Application (NDA) for ACER-001 (sodium phenylbutyrate) for oral suspension for the treatment of patients with urea cycle disorders (UCDs) and designated the NDA as a Class 2 resubmission. The FDA set a Prescription Drug User Fee Act (PDUFA) target action date of January 15, 2023.
- 18 Jul 2022 According to an Acer Therapeutics media release, additional existing nonclinical information as requested by the FDA in the CRL as well as labeling and other routine updates to the original NDA, were provided in the resubmission of the NDA.