Kebilidi is the first approved gene therapy that can be directly administered to the brain. Its OK secures a priority review voucher for PTC.

This therapy is designed to address the needs of children and adults with aromatic L-amino acid decarboxylase (AADC) deficiency. The FDA's accelerated approval is based on the saf

The FDA issued its first stamp of approval for a cell or gene therapy back in 2017 to Novartis' Kymriah. | The gene therapy has already been approved to treat AADC deficiency in Europe and the U.K., among other countries,

PTC Therapeutics (NASDAQ:PTCT) has received FDA approval for its gene replacement therapy Kebilidi for the treatment of children and adults with AADC deficiency, a rare genetic disorder that impairs the ability to generate dopamine,

PTC Therapeutics' Kebilidi gains FDA accelerated approval as the first brain-administered gene therapy in the U.S. for AADC deficiency treatment.

Kebilidi is the first gene therapy approved by the FDA for administration directly into the brain, given via a cannula during a surgical procedure, and replaces the human dopa decarboxylase (DDC) gene that is mutated in the disease, allowing dopamine synthesis to take place.