Eladocagene exuparvovec for the treatment of Aromatic L-amino acid decarboxylase (AADC): Regulatory Update
Highlights - Eladocagene exuparvovec is an investigational drug for the treatment of Aromatic L-amino acid decarboxylase (AADC) - Developed by PTC Therapeutics - Currently under EMA Review, the MMA application, decision delayed due to COVID - Decision Expected by Q2 2020 - Planned BLA submission to FDA by H1 2021 Aromatic L-amino acid decarboxylase (AADC) AADC deficiency is a rare genetic condition caused by a mutation in the dopa decarboxylase (DDC) gene, resulting in a lack of functioning AADC enzyme, which is responsible for the final step in the synthesis of key neurotransmitters dopamine and serotonin. AADC deficiency results in delays or failure to reach developmental milestones such as head control, sitting, standing, walking, or talking, low muscle tone (also known as muscular hypotonia), severe, seizure-like episodes involving involuntary eye movement (also known as oculogyric crises), autonomic abnormalities, and the need for life-long care. Given this neurologically dev...