On 31 January the FDA approved Ravicti (glycerol phenylbutyrate) from Hyperion Therapeutics, for the chronic management of some Urea Cycle...
Acer Therapeutics Inc. announced the resubmission of Acer’s New Drug Application (NDA) to the FDA for ACER 001 (sodium phenylbutyrate) for oral suspension for the treatment of patients with urea cycle disorders (UCDs).
Acer Therapeutics Inc. and its collaboration partner, Relief Therapeutics Holding SA announced the FDA has accepted for review Acer’s resubmitted New Drug Application (NDA) for ACER 001 (sodium phenylbutyrate) for oral suspension for the treatment of patients with urea cycle disorders (UCDs).
Cytonet has announced that the European Medicines Agency (EMA) has validated its Marketing Authorization Application (MAA) seeking approval of its...
The Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion recommending a change to the terms of...
Acer Therapeutics Inc. and its collaboration partner, Relief Therapeutics Holding SA announced that the FDA has issued a Complete Response Letter (CRL) regarding the New Drug Application (NDA) for ACER 001 (sodium phenylbutyrate) for oral suspension for the treatment of patients with urea cycle disorders (UCDs).
This was a long-term safety study HPN-100 in urea cycle disorder (UCD) subjects. Subjects were assessed regularly for safety and...
Urea cycle disorders (UCDs) are inborn errors of ammonia detoxification/arginine synthesis due to defects affecting the catalysts of the Krebs-Henseleit cycle (five core enzymes, one activating enzyme and one mitochondrial ornithine/citrulline antiporter) with an estimated incidence of 1:8.000.
Protocol HPN-100-005 was the first study of HPN-100 in pediatric subjects with urea cycle disorders (UCDs) and was a fixed-sequence,...
Horizon Pharma plc announced that it has submitted a supplemental New Drug Application (sNDA) with the FDA to expand the...