BioPharma Dive16h
PTC wins US approval of gene therapy for fatal enzyme disorder
Kebilidi is the first approved gene therapy that can be directly administered to the brain. Its OK secures a priority review ...
Monthly Prescribing Reference17h
Kebilidi, a Gene Therapy for AADC Deficiency, Gets Accelerated Approval
The Food and Drug Administration (FDA) has granted accelerated approval to Kebilidi™ (eladocagene exuparvovec-tneq) for the treatment of adult and pediatric patients with aromatic L-amino acid ...
GlobalData on MSN23h
FDA grants accelerated approval for PTC’s AADC deficiency gene therapy
This therapy is designed to address the needs of children and adults with aromatic L-amino acid decarboxylase (AADC) ...
Pharmabiz10h
PTC Therapeutics’ gene therapy, Kebilidi to treat AADC deficiency receives US FDA approval
PTC Therapeutics’ gene therapy, Kebilidi to treat AADC deficiency receives US FDA approval: Warren, New Jersey Friday, November 15, 2024, 10:00 Hrs [IST] PTC Therapeutics, Inc., ...
Business Insider21h
PTC Therapeutics Gains Buy Rating Following FDA Approval of Groundbreaking Gene Therapy Kebilidi
The recent FDA approval of Kebilidi, a groundbreaking gene therapy for AADC deficiency, is a significant milestone for the company. It is the first gene therapy directly administered to the brain and ...
US FDA approves PTC Therapeutics' gene therapy for ultra-rare disorder
The U.S. Food and Drug Administration approved PTC Therapeutics' gene therapy to treat a potential fatal enzyme deficiency ...
Managed Healthcare Executive1d
FDA Approves Gene Therapy for Debilitating Enzyme Deficiency
Kebilidi is a one-time gene therapy administered directly to the brain in patients with AADC deficiency, rare genetic ...
pharmaphorum1d
PTC gets FDA okay for first brain-delivered gene therapy
Kebilidi is the first gene therapy approved by the FDA for administration directly into the brain, given via a cannula during ...
BioSpace1d
PTC Wins First FDA Nod for Direct-to-Brain Gene Therapy, Targets Ultrarare Disease
The FDA has followed in the footsteps of its European counterparts and granted accelerated approval to PTC Therapeutics’ gene ...
precisionmedicineonline21h
PTC Therapeutics' AADC Deficiency Gene Therapy Kebilidi Gains FDA Accelerated Approval
This is the first gene therapy for AADC deficiency to receive US regulatory approval and the first that can be administered directly to the brain.
BioWorld1d
PRV sweetens PTC approval of Kebilidi in AADC deficiency
PTC Therapeutics Inc.’s accelerated go-ahead from the U.S. FDA for Kebilidi (eladocagene exuparvovec-tneq), a recombinant adeno-associated virus serotype 2-based gene therapy to treat aromatic L-amino ...