Kebilidi, a Gene Therapy for AADC Deficiency, Gets Accelerated Approval. Diana Ernst, RPh | Publish Date November 14, 2024 . Kebilidi is a one-time, recombinant adeno-associated virus- ...

FDA grants accelerated approval to PTC Therapeutics' Kebilidi, the first brain-administered gene therapy in the U.S. for AADC deficiency. Clinical trials show Kebilidi improved motor function in 8 ...

Kebilidi is the first FDA-approved gene therapy for the deficiency. PTC shares gained around 8.5 percent in the extended trading on the Nasdaq on Thursday, at $45.97.

The PTC Therapeutics gene therapy, Kebilidi, treats an enzyme deficiency that affects the body’s ability to produce dopamine, a neurotransmitter important for motor control. FDA approval for the ...

T he US Food and Drug Administration (FDA) has granted accelerated approval for PTC Therapeutics’ KEBILIDI, the first gene therapy in the US that is directly delivered to the brain.

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 ...

PTC Therapeutics’ Kebilidi™ (US) / Upstaza™ (ex-US) (eladocagene exuparvovec), an AAV vector-based gene therapy indicated for adults and children with aromatic12 L-amino acid decarboxylase ...

The gene therapy, which will be marketed in the United States with the brand name Kebilidi (eladocagene exuparvovec-tneq), is indicated for the treatment of children and adults with aromatic L ...