The FDA requested a Endocrinologic and Metabolic Drugs Advisory Committee (EMDAC) for the review of Carbaglu which is currently in the process of New Drug Approval (NDA) review in the USA. The EMDAC held their meeting on 13th January 2010 in which the efficacy, potential side effects and overall risk/benefit profile of Carbaglu were discussed. The EMDAC was very positive and notably the vote was unanimous for its risk/benefit profile.
This is an important step towards the NDA approval which will provide great benefit to patients with NAGS deficiency and their families.
Carbaglu® (carglumic acid) currently has marketing authorization in Europe since 2003 for the treatment of N-Acetyl Glutamate Synthase (NAGS) deficiency (more information about
NAGS).