FDA Approves First Gene Therapy for Rare Genetic Disorder AADC Deficiency

The U.S. Food and Drug Administration (FDA) has approved Kebilidi (eladocagene exuparvovec-tneq), a groundbreaking gene therapy for the treatment of aromatic L-amino acid decarboxylase (AADC) deficiency. This approval marks a historic moment, as Kebilidi becomes the first-ever gene therapy authorized by the FDA for this rare and debilitating condition.

Dr. Peter Marks, M.D., Ph.D., director of the FDA’s Center for Biologics Evaluation and Research (CBER), praised the approval, noting, “Clinical advancements in gene therapy are continually unveiling innovative treatments for rare diseases that have long been challenging to manage. Today’s approval reinforces our dedication to making safe, effective treatments accessible to those who need them most.”

AADC deficiency is a rare genetic disorder that disrupts the production of key neurotransmitters, which are crucial for proper communication within the nervous system. Individuals with AADC deficiency often face significant challenges, including delays in gross motor function (such as head control, sitting, and walking), hypotonia (low muscle tone), and developmental as well as cognitive delays.

SOURCE: https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapy-treatment-aromatic-l-amino-acid-decarboxylase-deficiency 

CREDITS: U.S. FOOD AND DRUG ADMINISTRATION