Gene Therapy for AADC Deficiency Gets FDA’s Priority Review

The Food and Drug Administration (FDA) has accepted for Priority Review the Biologics License Application (BLA) for eladocagene exuparvovec (Upstaza^™), a gene therapy for the treatment of aromatic L–amino acid decarboxylase (AADC) deficiency.

AADC deficiency is a rare genetic disorder characterized by decreased activity of the AADC enzyme, which is responsible for the synthesis of neurotransmitters dopamine and serotonin. The disease typically causes severe disability starting from the first months of life and affects physical, mental, and behavioral functions.

Eladocagene exuparvovec is a one-time, recombinant adeno-associated virus serotype 2 (AAV2)-based gene replacement therapy. It is designed to deliver a functioning human dopa decarboxylase (DDC) gene directly into the putamen, thereby increasing the AADC enzyme and restoring dopamine production.

The BLA is supported by efficacy and safety data from clinical trials and compassionate use programs, which demonstrated neurological improvements in patients with AADC deficiency. Findings from a long-term study showed rapid improvement in motor and cognitive function that was sustained through more than 5 years of follow up.

“We are excited to be one step closer to bringing an approved therapy to patients with AADC deficiency in the United States,” said Matthew B. Klein, MD, CEO, PTC Therapeutics. “The data collected to date continue to support the transformative benefit of Upstaza, this highly innovative gene therapy directly infused into the brain.”

A Prescription Drug User Fee Act target date of November 13, 2024 has been set for the application.

This article originally appeared on MPR