The Duchenne Muscular Dystrophy (DMD) community has received disappointing news as Pfizer Inc. announced the Phase 3 CIFFREO study of fordadistrogene movaparvovec, an investigational mini-dystrophin gene therapy, did not meet its primary endpoint. This study, which was closely watched by families and medical professionals alike, aimed to demonstrate improved motor function in ambulatory boys aged 4 to 7 with DMD. The results, measuring the change in the North Star Ambulatory Assessment (NSAA) after one year of treatment compared to placebo, unfortunately did not show significant improvement.
Adding to the setback, key secondary endpoints including 10-meter run/walk velocity and time to rise from floor velocity also failed to show a significant difference between the treated group and the placebo group. This outcome is a stark reminder of the complexities in developing effective treatments for DMD, a severe genetic disorder.
Pfizer has stated its commitment to transparency and ongoing research, planning to share detailed findings from the CIFFREO study at upcoming medical and patient advocacy forums, including the PPMD’s 30th Annual Conference. This move underscores Pfizer’s dedication to contributing to the collective knowledge base and fostering advancements in DMD treatment development, even in the face of clinical trial disappointments. The company aims to ensure that lessons learned from this trial are used to refine future research and development strategies for Duchenne therapies.
Despite this disheartening outcome, Pfizer has affirmed its continued monitoring of all participants enrolled in the CIFFREO study. The company is currently evaluating the next steps for the fordadistrogene movaparvovec program, emphasizing their long-term commitment to the DMD community. This dedication reflects a broader approach of “sharing the care,” where Pfizer’s support extends beyond just drug development to encompass ongoing patient support and community engagement.
The Parent Project Muscular Dystrophy (PPMD) expressed its deep disappointment at the study results, acknowledging the significant impact on the DMD community and especially those who participated in the trial. PPMD also highlighted the crucial role of patients and families who participate in clinical trials, recognizing their invaluable contributions to advancing medical science, regardless of immediate benefits. Their bravery and commitment are essential to the ongoing pursuit of effective treatments.
In alignment with the spirit of “sharing the care,” both Pfizer and PPMD reiterated their ongoing partnership and commitment to the DMD community. PPMD will continue its collaborative efforts with researchers, industry partners, and the wider Duchenne community to accelerate the development of safe and effective treatments. The focus remains steadfast on bringing hope and tangible therapeutic options to individuals and families affected by DMD. The resilience of the DMD community, coupled with the persistent efforts of organizations like Pfizer and PPMD, are crucial in navigating the challenges and continuing the journey towards better treatments and improved lives.
For more detailed information, please refer to Pfizer’s press release here and Pfizer’s community letter.
