FDA Approves First Gene Therapy for Treatment of Aromatic L …
1 day ago · The U.S. Food and Drug Administration approved Kebilidi (eladocagene exuparvovec-tneq), an adeno-associated virus vector-based gene therapy indicated for the treatment of adult and pediatric ...
PTC wins US approval of gene therapy for fatal enzyme disorder
23 hours ago · PTC Therapeutics on Wednesday won Food and Drug Adminitration approval for Kebilidi, the first gene therapy cleared in the U.S. for direct administration to the brain. The treatment is designed for patients with aromatic L-amino acid decarboxylase, or AADC, deficiency, a condition that affects the way neurons transmit information to other cells.
FDA approves Kebilidi as first gene therapy to treat aromatic L …
1 day ago · The first gene therapy for for the treatment of aromatic L-amino acid decarboxylase (AADC) deficiency for adults and pediatric patients, eladocagene exuparvovec-tneq (Kebilidi; PTC Therapeutics), has been approved by the FDA according to an announcement from the federal agency. 1 AADC deficiency is a rare genetic disorder that affects production of neurotransmitters that are chemical ...
PTC wins FDA approval for first brain-delivered gene therapy Kebilidi
23 hours ago · 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, a Gene Therapy for AADC Deficiency, Gets Accelerated
23 hours ago · Kebilidi, a Gene Therapy for AADC Deficiency, Gets Accelerated Approval. Kebilidi is a one-time, recombinant adeno-associated virus-based gene therapy administered through intraputaminal infusion ...
PTC Therapeutics Announces FDA Approval of AADC Deficiency Gene Therapy
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 AADC deficiency, including the full spectrum of disease severity. Launch preparations are well underway, with centers of excellence already identified and ...
PTC Wins First FDA Nod for Direct-to-Brain Gene Therapy, Targets ...
1 day ago · It is the first approved gene therapy to be delivered directly to the brain. The FDA on Wednesday signed off on PTC Therapeutics’ gene therapy eladocagene exuparvovec-tneq for the treatment of aromatic L-amino acid decarboxylase deficiency. The treatment will be marketed under the brand name Kebilidi. With only around 350 cases reported in ...
KEBILIDI | FDA - U.S. Food and Drug Administration
1 day ago · (1, 14) KEBILIDI is an adeno-associated virus (AAV) vector-based gene therapy indicated for the treatment of adult and pediatric patients with aromatic L amino acid decarboxylase (AADC) deficiency.
US FDA approves PTC Therapeutics' gene therapy for ultra-rare …
1 day ago · The therapy, sold under the brand name Kebilidi, is a one-time treatment for children and adults with an ultra rare inherited disorder called aromatic l-amino acid decarboxylase (AADC) deficiency.
FDA grants accelerated approval for PTC’s AADC deficiency gene therapy
1 day ago · Credit: Gatot Adri via Shutterstock. The 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. This therapy is designed to address the needs of children and adults with aromatic L-amino acid decarboxylase (AADC) deficiency.
- Some results have been removed