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- ...

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 ...

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 ...

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.

The gene therapy, marketed under the brand name Kebilidi, is the first in the United States to be injected directly into the brain. It is approved for children with fully developed skulls and for ...

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.

Orsini announced today that it has been chosen by PTC Therapeutics as the exclusive specialty pharmacy provider for the adeno-associated virus vector-based gene therapy KEBILIDI™ (eladocagene ...

The U.S. Food and Drug Administration said on Tuesday that it is investigating reports of two deaths due to acute liver ...

In a note to clients, analysts at William Blair hailed the approval as a positive development for the wider gene therapy space and modeled peak Kebilidi revenue of $266.3 million in 2026.

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 ...

In one of the first major tests of the new FDA leadership’s regulatory philosophy toward gene therapies for rare diseases, ...