Pfizer Inc. said a third patient had to be hospitalized after being treated with the company’s experimental gene therapy for a rare degenerative muscular condition, but said the safety profile still supports launching a larger late-stage trial of the treatment.
Pfizer Inc. is investing $500 million to expand a manufacturing facility in Sanford, North Carolina, that plays a central role in the company’s efforts to become a major player in gene therapy.
Sarepta Therapeutics Inc. was informed that an adverse event report was erroneously submitted to the U.S. health regulator regarding an ongoing study of the company’s gene therapy for Duchenne muscular dystrophy (DMD).
Research from three clinical trials supports that Sarepta Therapeutics’ Exondys 51 slows respiratory decline in DMD.
Pfizer’s IV Revatio, when added to inhaled nitric oxide, failed to meet the primary efficacy endpoint in treating newborns with persistent pulmonary hypertension in a Phase III study.