Pfizer Provides Update on Phase 3 Study of Investigational Gene Therapy for Ambulatory Boys with Duchenne Muscular Dystrophy
The overall safety profile of fordadistrogene movaparvovec in the CIFFREO trial was manageable, with mostly mild to moderate adverse events, and treatment-related serious adverse events generally responding to clinical management.
“We are extremely disappointed that these results did not demonstrate the relative improvement in motor function that we had hoped. We plan to share more detailed results from the study at upcoming medical and patient advocacy meetings, with the goal of ensuring that learnings from this trial can help improve future clinical research and development of treatment options that can improve care for boys living with Duchenne muscular dystrophy,” said
About the Fordadistrogene Movaparvovec Clinical Program
CIFFREO is a Phase 3 global, multi-center, randomized, double-blind, placebo-controlled study to assess the safety and efficacy of fordadistrogene movaparvovec investigational gene therapy in ambulatory male participants, aged 4 to 7 years, with a genetic diagnosis of DMD who are on a stable daily regimen of glucocorticoids. The primary endpoint of the study is a change from baseline to one year in the North Star Ambulatory Assessment (NSAA) total score. For more information, visit ciffreoduchennetrial.com or clinicaltrials.gov.
The CIFFREO study is currently on a dosing pause due to a fatal serious adverse event in the Phase 2 DAYLIGHT trial (NCT05429372). DAYLIGHT is a study that is evaluating the safety and tolerability of fordadistrogene movaparvovec in participants 2 years to 3 years of age with DMD.
About Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (DMD) is a serious genetic disease characterized by progressive muscle degeneration and weakness. Symptoms usually manifest in early childhood between the ages of 3 and 5. The disease primarily affects boys. Muscle weakness can begin as early as age 3, first affecting the muscles of the hips, pelvic area, thighs, and shoulders, and later the skeletal (voluntary) muscles in the arms, legs, and trunk. By their early teens, patients typically lose their ability to walk and the heart and respiratory muscles are also affected, ultimately resulting in premature death. DMD is the most common form of muscular dystrophy worldwide with an incidence of 1 in every 5,000 live male births.1
About
At
Disclosure Notice
The information contained in this release is as of
This release contains forward-looking information about fordadistrogene movaparvovec, an investigational mini-dystrophin gene therapy for Duchenne muscular dystrophy, topline results from the Phase 3 CIFFREO study and plans to share more detailed results from the study at upcoming medical and patient advocacy meetings that involve substantial risks and uncertainties that could cause actual results to differ materially from those expressed or implied by such statements. Risks and uncertainties include, among other things, the uncertainties inherent in research and development; uncertainties related to further analysis of data from the Phase 3 CIFFREO study, including the sharing of more detailed results from the study at upcoming medical and patient advocacy meetings; uncertainties regarding the commercial success of Pfizer’s gene therapy portfolio; uncertainties regarding the impact of COVID-19 on Pfizer’s business, operations and financial results; and competitive developments.
A further description of risks and uncertainties can be found in Pfizer’s Annual Report on Form 10-K for the fiscal year ended
1 Crisafulli S, Sultana J, Fontana A, Salvo F, Messina S, Trifirò G. Global epidemiology of Duchenne muscular dystrophy: an updated systematic review and meta-analysis. Orphanet J Rare Dis. 2020;15(1):141.
View source version on businesswire.com: https://www.businesswire.com/news/home/20240612445038/en/
Media:
+1 (212) 733-1226
PfizerMediaRelations@pfizer.com
Investor:
+1 (212) 733-4848
IR@pfizer.com
Source: