26 January 2021 - Carbaglu is first and only FDA approved medication for hyperammonaemia associated with these rare conditions.
Recordati Rare Diseases today announced the U.S. FDA has approved a new indication for Carbaglu (carglumic acid) 200 mg tablets as adjunctive therapy to standard of care for the treatment of acute hyperammonaemia due to propionic acidaemia (PA) or methylmalonic acidaemia (MMA) in paediatric and adult patients.
FDA approval of the new indication was supported by a randomised, double-blind, placebo-controlled, multi-centre clinical trial comparing the effectiveness of Carbaglu to placebo in the treatment of hyperammonaemic episodes in patients with PA or MMA.