CIFFERO clinical trial for DMD gene therapy cancelled

31 July 2024

In June, we shared news that Pfizer’s CIFFERO phase 3 clinical trial for Duchenne muscular dystrophy (DMD) gene therapy failed to meet its primary and secondary objectives. We are sad to inform you that the trial has now been cancelled.

The CIFFREO Phase 3 clinical trial was a global study that aimed to test if the gene therapy fordadistrogene movaparvovec was safe and effective for use in boys with DMD aged four to seven. Fordadistrogene movaparvovec was a potential gene therapy that delivers a version of dystrophin gene, called mini dystrophin, into muscle cells. People with DMD lack a protein called dystrophin, which is required to maintain the strength of muscles.

While we are disappointed that the work on fordadistrogene movaparvovec won’t be going ahead, all boys who received the gene therapy will continue to be monitored, with their safety being the main priority.

Pfizer continues to review the results of the trial collected so far and will be sharing updates at future scientific meetings.

If you or your family member has been involved in the study, we recommend that you contact the study site for any further information.