PTC Therapeutics Announces FDA Approval of AADC Deficiency …
1 day ago · Indication. KEBILIDI is an adeno-associated virus vector-based gene therapy indicated for the treatment of adult and pediatric patients with aromatic L-amino acid decarboxylase (AADC) deficiency ...
PTC Therapeutics Announces FDA Approval of AADC Deficiency …
1 day ago · PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today the U.S. Food and Drug Administration (FDA) accelerated approval of its gene therapy for the treatment of AADC deficiency, the first-ever gene ...
FDA grants accelerated approval for PTC’s AADC deficiency gene …
12 hours 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 wins FDA approval for first brain-delivered gene therapy Kebilidi
11 hours ago · The FDA's Kebilidi approval covers children and adults with AADC deficiency across the “full spectrum” of disease severity, according to PTC. In a note to clients, analysts at William Blair ...
PTC Therapeutics’ AADC deficiency gene therapy granted FDA …
1 day ago · Nov. 13, 2024, 05:10 PM. PTC Therapeutics (PTCT) announced that the FDA accelerated approval of its gene therapy for the treatment of AADC deficiency, the first-ever gene therapy approved in the U ...
FDA Grants Accelerated Approval to PTC Therapeutics’ Gene …
2 days ago · According to a new announcement, the FDA has granted PTC Therapeutics’ gene therapy eladocagene exuparvovec-tneq an accelerated approval indicated for the treatment of patients aged 18 years and older with aromatic L-amino acid decarboxylase (AADC) deficiency, including the full spectrum of disease severity. 1 Marketed under Kebilidi, it becomes the first-ever gene therapy that is directly ...
FDA Approves First Gene Therapy for Treatment of Aromatic L …
1 day ago · “AADC deficiency can cause a range of debilitating symptoms, including life-threatening complications,” said Nicole Verdun, M.D., director of the Office of Therapeutic Products in CBER.
PTC Therapeutics Announces FDA Acceptance and Priority Review …
2 days ago · WARREN, N.J., May 14, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today that the FDA has accepted for filing the Biologics License Application (BLA) for Upstaza™ (eladocagene exuparvovec), a …
PTC wins US approval of gene therapy for fatal enzyme disorder
11 hours ago · Dive Insight: The approval is welcome news for PTC, which has struggled with a number of setbacks that dented its share price. PTC stock climbed above $58 in May 2023, just before the company announced a Phase 3 study failure for a Friedreich ataxia drug. By Nov. 2023, after a negative regulatory decision for a Duchenne muscular dystrophy medicine in September, the shares were trading below $19.
PTC now seeking US approval of one-time gene therapy Upstaza - AADC News
Mar 20, 2024 · PTC Therapeutics has submitted its application seeking the approval of Upstaza (eladocagene exuparvovec) — a one-time gene therapy to correct the underlying cause of aromatic L-amino acid decarboxylase (AADC) deficiency — to the U.S. Food and Drug Administration (FDA).
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