PTCT · PTC Therapeutics, Inc.

Summary

PTC Therapeutics, Inc. withdrew its New Drug Application resubmission for Translarna (ataluren) for nonsense mutation Duchenne muscular dystrophy following U.S. Food and Drug Administration feedback that the data are unlikely to meet the threshold of substantial evidence of effectiveness. The press release is furnished as Exhibit 99.1 and not deemed filed under Section 18 of the Exchange Act.

Key takeaways

• PTC Therapeutics withdrew NDA resubmission for Translarna (ataluren) on February 12, 2026
• Withdrawal followed FDA feedback on application review
• FDA indicated NDA data unlikely to meet substantial evidence threshold for approval
• Translarna targets nonsense mutation Duchenne muscular dystrophy
• Press release issued from Warren, N.J., furnished as Exhibit 99.1
• Matthew B. Klein, M.D., Chief Executive Officer, commented on the decision