SOUTH PLAINFIELD, N.J. - PTC Therapeutics, Inc. (NASDAQ: NASDAQ:PTCT) announced today the submission of a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for Upstaza™ (eladocagene exuparvovec), a gene therapy aimed at treating aromatic L-amino acid decarboxylase (AADC) deficiency.
The company also disclosed plans to re-submit a New Drug Application (NDA) for Translarna™ (ataluren) targeting nonsense mutation Duchenne muscular dystrophy (nmDMD) following recent FDA feedback, with the submission expected by mid-year.
Matthew B. Klein, M.D., Chief Executive Officer of PTC (NASDAQ:PTC) Therapeutics, expressed optimism about the submissions, highlighting the potential benefits of Upstaza for children with the severe genetic disorder AADC deficiency. He also noted the anticipation for the Translarna NDA resubmission, recognizing the needs of U.S. patients and families affected by nmDMD.
Upstaza is a one-time gene replacement therapy designed for patients aged 18 months and older with a confirmed diagnosis of AADC deficiency. The therapy, administered via a neurosurgical procedure, has shown transformational neurological improvements in clinical trials and compassionate use programs. Side effects reported include insomnia, irritability, and dyskinesia. Upstaza has already been authorized in Europe, Great Britain, and Israel.
AADC deficiency is a rare and typically fatal genetic disorder that severely impacts children from the first months of life, often requiring ongoing therapy and interventions to manage life-threatening complications.
Translarna, developed by PTC Therapeutics, is a protein restoration therapy for genetic disorders caused by a nonsense mutation, such as nmDMD, a rare and fatal genetic disorder leading to progressive muscle weakness and premature death.
PTC Therapeutics is a global biopharmaceutical company focused on the development and commercialization of medicines for rare disorders. The company's pipeline and scientific expertise aim to address the needs of patients with limited treatment options.
This announcement is based on a press release statement from PTC Therapeutics. The company's forward-looking statements involve risks and uncertainties, including the potential outcomes of regulatory submissions and the commercialization of its products. There are no guarantees of regulatory approval or commercial success for Upstaza or Translarna.
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