Pfizer DMD trial moves forward after FDA lifts hold

pharmafile | April 29, 2022 | News story | Medical Communications  

Pfizer has announced that it will open the first ever US sites in its Phase III trial study, evaluating its dystrophin gene therapy in ambulatory patients with Duchenne muscular dystrophy (DMD).

The company announcement comes four months after the unexpected death of a patient during Pfizer’s screening and dosing of a Phase Ib study, which led to the trial being paused.

The FDA has since lifted its clinical hold on the Investigational New Drug (IND) application for fordadistrogene movaparvovec. Pfizer has addressed the agency’s requests related to the potency assay.

The protocol amendments include a seven-day hospitalisation period, to enable closer monitoring and management of patients following the administration of gene therapy.

The Phase III study, Ciffreo, has been ongoing in 11 countries, with regulatory authorities in the UK, Canada, Taiwan, Spain, and Belgium approving the re-start of the Phase III study.

Duchenne muscular dystrophy is an inherited disorder characterised by progressive muscular weakness. It is caused by mutations in the gene encoding dystrophin, which is a protein needed by the muscles for stability. Children with DMD will have difficulty standing and walking.  

“Duchenne muscular dystrophy is a devastating disease with very limited treatment options, and we believe that gene therapy has the potential to significantly impact disease progression,” Brenda Cooperstone, Chief Development Officer of Pfizer’s rare disease global product development, said. 

“Pfizer is pleased to progress Ciffreo and is working as quickly as possible to activate trial sites as local regulatory and ethics approvals occur. We thank the participants in our clinical trials and their families, as well as the broader Duchenne community, for their ongoing trust and collaboration as we work to advance our investigational gene therapy,” Cooperstone added. 

Lina Adams

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