FDA Lifts Clinical Hold on Pfizer DMD Gene Therapy Linked to Patient Death
Pfizer said today it will open its first U.S. sites for a global Phase III trial assessing its fordadistrogene movaparvovec (PF-06939926) in Duchenne muscular dystrophy (DMD) after the FDA lifted a clinical hold on the company’s investigational new drug (IND) application for the program, which was imposed in December 2021 after a patient died in a Phase Ib open-label study of the mini-dystrophin gene therapy candidate.
The global Phase III CIFFREO trial (NCT04281485), designed to evaluate the safety and efficacy of fordadistrogene movaparvovec in boys with DMD, has been ongoing in 11 countries. It was paused in August 2021 following three “severe adverse events of muscle weakness, two of which involved myocarditis (inflammation of the heart tissue),” Pfizer said at the time. The trial was resumed in November after a protocol amendment, before being paused again the following month after the patient death.
To date, regulators in Belgium, Canada, Spain, Taiwan, and the United Kingdom have approved the re-start of the Phase III trial, with additional global reviews ongoing. Pfizer anticipates that nearly all CIFFREO sites will open by the end of June.
“Pfizer is pleased to progress CIFFREO and is working as quickly as possible to activate trial sites as local regulatory and ethics approvals occur,” Brenda Cooperstone, Chief Development Officer, Rare Disease, Pfizer Global Product Development, said in a statement.
Shares of Pfizer inched up nearly 2% in trading today, to $50.55 from yesterday’s closing price of $49.74.
https://www.genengnews.com/topics/genome-editing/gene-therapy/fda-lifts-clinical-hold-on-pfizer-dmd-gene-therapy-linked-to-patient-death/
ارسال به دوستان