PTC Jumps as Drugmaker Says Therapy May Help Some DMD Patients

  • Patients on ataluren had better breathing than historical set
  • DMD is a deadly muscle disease that affects young boys

PTC Therapeutics Inc. jumped in early market trading after saying an experimental therapy for a deadly muscle disease, which was rejected by U.S. regulators earlier this year, may have helped some patients preserve lung function in a study.

Patients with Duchenne muscular dystrophy who took the drug, called ataluren, may have had their ability to breathe preserved when compared against a previous study assessing patients’ pulmonary functions, PTC said in a statement Thursday. Comparing patients in a study to those observed in other settings is not considered the highest quality evidence for a drug’s effectiveness.

PTC rose 6.5 percent to $14.60 at 8:58 a.m. in New York before markets opened.

PTC is looking to come back after the U.S. Food and Drug Administration in February refused to review its drug application for ataluren, deeming it incomplete. Hopes for PTC’s eventual success have risen since a competitor, Sarepta Therapeutics Inc., had its drug for a form of DMD approved in September on the basis of a development program the agency’s director called “seriously flawed.” Since that approval PTC’s shares had risen 50 percent through Wednesday’s close.

PTC is now working through a dispute resolution process with the FDA on its submission for ataluren, spokeswoman Jane Baj said. The company hopes “to have more visibility on what the next steps are in the coming months,” she said.

DMD is a rare, genetic muscle disease that affects young boys and usually kills them in their youth.

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