PTC Therapeutics (PTCT) Provides Regulatory Updates

PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced the submission of a BLA to the U.S. FDA for Upstaza™ (eladocagene exuparvovec), a gene therapy for the treatment of aromatic L-amino acid decarboxylase (AADC) deficiency. In addition, based on recent feedback from the FDA, PTC plans to re-submit an NDA for Translarna™ (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD). PTC expects to make the NDA resubmission by mid-year.

"We are excited to bring Upstaza one step closer to children in the United States with the highly mortal and fatal disease of AADC deficiency. The data collected to date continue to demonstrate the transformative clinical benefits the gene therapy provides to patients," said Matthew B. Klein, M.D., Chief Executive Officer of PTC Therapeutics. "In addition, we look forward to the Translarna NDA resubmission this summer. We know U.S. patients and families have been waiting for an approved therapy that specially targets nmDMD."

In addition, the sepiapterin MAA for PKU remains on schedule for submission to the EMA by the end of this month.

You May Also Be Interested In

• ARS Pharmaceuticals cuts 2026 expenses after no new neffy coverage gains
• Ollin Biosciences raises $330M Series B for eye disease drug trials
• Viking Therapeutics starts Phase 1 trial of weight loss drug VK3019