Pfizer announced today that enrollment is now complete for both the Phase 3 CIFFERO study and Phase 2 DAYLIGHT study of PF-06939926 (fordadistrogene movaparvovec) in Duchenne.

As always, we are so grateful to all of the families who participate in clinical trials.

We look forward to continued updates and hearing more from Pfizer at PPMD’s 2023 Annual Conference in Dallas, TX, June 29- July 1.

Read the Update From Pfizer:

April 21st, 2023 

Thanks to the overwhelming response of the patient community, we are pleased to announce today that participant screening for the CIFFREO trial, our global phase 3 study evaluating the investigational mini-dystrophin gene therapy, fordadistrogene movaparvovec, in ambulatory patients with Duchenne muscular dystrophy (DMD), is now complete. We would like to express our sincere gratitude to the enthusiasm and trust from participants and their families, as well as the tireless efforts of investigators and study teams who recruited participants across 40 sites in 15 countries. 

This will be reflected in the upcoming weeks in the clinical trials status on clinicaltrials.gov: https://www.clinicaltrials.gov/ct2/show/NCT04281485 

We are also pleased to share that participant identification in Pfizer’s open-label DAYLIGHT trial, which is enrolling approximately ten 2-3 year-old ambulatory patients, across 6 clinical trial sites in Australia and the United States, has been completed as well: 

https://www.clinicaltrials.gov/ct2/show/NCT05429372 

We will continue to engage with regulatory authorities and are focused on bringing this potential important treatment option to patients with DMD as quickly and safely as possible. Overall, we are optimistic that our gene therapy program will generate robust clinical data on fordadistrogene movaparvovec. Pending study results and regulatory review, we are fully committed to delivering a much-needed potential new treatment option to patients with DMD. Since the beginning of this program, we have shared results and regular updates with the Duchenne community whenever available, including preliminary data from our phase 1b: 

One year data from ambulatory boys in a phase 1b, open-label study of fordadistrogene movaparvovec (PF-06939926) for Duchenne muscular dystrophy (DMD) – MDA Clinical & Scientific Conference 2022 (mdaconference.org) 

We will continue to share information with the Duchenne community as it becomes available. As always, we want to express our appreciation for your trust and collaboration as this gene therapy program for boys with Duchenne muscular dystrophy continues to advance. We also want to thank once again the participants in our clinical program and their families, as well as our investigators, without whom the development of this much-needed potential new therapy would not be possible. 

Sincerely, 

The Pfizer DMD gene therapy team