Home » Pfizer Signs Up First UK Participant in Late-Stage Study of DMD Gene Therapy
Pfizer Signs Up First UK Participant in Late-Stage Study of DMD Gene Therapy
Drugs Regulatory Affairs
Pfizer has enrolled the first British participant in a phase 3 study evaluating its experimental gene therapy PF-06939926 in boys with Duchenne muscular dystrophy (DMD).
The trial, which will extend to 55 sites in 15 countries, plans to enroll 99 male patients from the ages of four to seven, the company said.
The FDA granted PF-06939926 Fast-Track designation in October 2020. The experimental gene therapy received the agency’s Orphan Drug and Rare Pediatric Disease designations in May 2017.