FDA Approves First Gene Therapy for Treatment of Aromatic L …
1 day ago · The efficacy of Kebilidi was demonstrated based on gross motor function improvement in 8 of 12 treated patients, which has not been reported in untreated patients with the severe presentation of ...
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 ...
FDA grants accelerated approval for PTC’s AADC deficiency gene …
1 day ago · The FDA's accelerated approval is based on the safety and clinical efficacy findings from the ongoing PTC-AADC-GT-002 global clinical trial. KEBILIDI works by replacing the defective gene and is ...
PTC Therapeutics Announces FDA Approval of AADC Deficiency …
1 day ago · The company plans to monetize the voucher. Story Continues. PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today the U.S. Food and Drug Administration (FDA) accelerated approval of its gene ...
Kebilidi, a Gene Therapy for AADC Deficiency, Gets Accelerated
23 hours ago · Kebilidi is a one-time, recombinant adeno-associated virus-based gene therapy administered through intraputaminal infusion. ... News release. PTC Therapeutics. November 13, 2024.
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.
PTC Wins First FDA Nod for Direct-to-Brain Gene Therapy, Targets ...
1 day ago · 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 the literature, aromatic L-amino acid decarboxylase (AADC) deficiency is an ultrarare genetic disease characterized by severe ...
PTC gets FDA okay for first brain-delivered gene therapy
1 day ago · 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 ...
PTC Therapeutics' AADC Deficiency Gene Therapy Kebilidi Gains …
23 hours ago · The FDA-approved indication for Kebilidi is broad, allowing access to children and adults and patients across the full spectrum of disease severity, PTC emphasized in a statement. PTC is now preparing to commercially launch Kebilidi and said it has identified centers of excellence where surgeons are trained to administer the gene therapy.
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.
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