FDA Sets PDUFA Date for Gene Therapy Targeting AADC Deficiency

Key Insights

• The FDA has set an action date of April 29, 2025, for a Biologics License Application (BLA) for a gene therapy intended to treat Aromatic L-Amino Acid Decarboxylase (AADC) deficiency.
• The gene therapy, already approved in the UK and European Union under the brand name Upstaza since 2022, showed promising results in clinical trials.
• The pivotal phase 1b/2 FELIX clinical trial (NCT04404660) demonstrated good rates of overall complete remission and median duration of remission for a CAR-T therapy.

The U.S. Food and Drug Administration (FDA) has set a Prescription Drug User Fee Act (PDUFA) action date of April 29, 2025, for a Biologics License Application (BLA) concerning a gene therapy designed to treat Aromatic L-Amino Acid Decarboxylase (AADC) deficiency. This regulatory milestone marks a significant step toward potential approval in the United States for a therapy already available in other regions.

The gene therapy has been approved in the UK and European Union since 2022 under the brand name Upstaza. The therapy targets the underlying genetic defect in AADC deficiency, a rare and severe neurological disorder that affects the production of dopamine and serotonin.

CAR-T therapy was approved based on the pivotal phase 1b/2 FELIX clinical trial (NCT04404660), which showed good rates of overall complete remission and median duration of remission. The safety data set for the trial included 39 patients who were treated with ALLO-316.