PPMD is disappointed to learn that Pfizer Inc. has officially discontinued development of the company’s investigational mini-dystrophin gene therapy, fordadistrogene movaparvovec. This announcement follows Pfizer’s June 2024 update on the Phase 3 CIFFREO study evaluating the drug in ambulatory patients with Duchenne ages 4 to 7.

In June 2024, Pfizer shared that the study did not meet its primary endpoint of improvement in motor function among patients treated with the gene therapy compared to placebo. Pfizer’s Dan Levy, MD, later participated in the “Community Conversation With Pfizer” session at PPMD’s 30th Annual Conference, where he clarified that the CIFFREO study was not considered terminated at that time. Today, Pfizer indicated that despite the program’s now termination, the company is focused on conducting a more detailed review of these data, which will be shared at future medical and patient advocacy forums.

To ensure participant safety, Pfizer will be following up with all patients who received fordadistrogene movaparvovec in the clinical program for long-term safety monitoring. With this, it is important to note that drug development was not discontinued for safety reasons. This termination does, however, mean that Pfizer is not currently active in the Duchenne treatment space, which is a devastating setback for our community.

We want to acknowledge and thank the brave patients and families who participate in clinical trials, despite the absence of guaranteed benefit. Your contributions are invaluable in helping to bring therapies to all in our community.

PPMD will continue to work closely with researchers, industry partners, and the entire Duchenne community to ensure that every effort is made to bring safe and effective treatments to those who need them. 

Read Pfizer’s Patient Advocacy Group letter here:

July 30, 2024

Since last month’s announcement on the results from the Phase 3 CIFFREO study evaluating fordadistrogene movaparvovec, we have made the difficult decision to discontinue its development.

At this time we are focused on conducting a more detailed review of these data, which will be shared at future medical and patient advocacy forums. To ensure participant safety, all boys who received fordadistrogene movaparvovec in the clinical program will be followed up for long-term safety monitoring.

We are greatly saddened by the results, and the CIFFREO outcome is not what any of us hoped for. But we believe there will be much to learn from these findings and hope they can contribute to future research that could lead to future scientific breakthroughs for boys living with DMD.

We know how absolutely crucial it is to find transformative treatments for boys that are living with DMD and we want to again thank this incredible community – including but not limited to the participants who have enrolled in trials in the fordadistrogene movaparvovec program, their supportive families, and the trial investigators involved.

Sincerely,
The Pfizer DMD gene therapy team