Posted by Michael Wonder on 26 Jan 2021
Recordati Rare Diseases: Carbaglu (carglumic acid) receives U.S. FDA approval for a new indication to treat acute hyperammonaemia associated with propionic acidemia and methylmalonic acidemia
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.
