DTMT Network

Global pharmaceutical giant Pfizer has announced that the company is planning to open the first U.S. sites in the Phase 3 study evaluating the investigational mini-dystrophin gene therapy, fordadistrogene movaparvovec, in ambulatory patients with Duchenne muscular dystrophy (DMD).

The announcement came after the lifting of the clinical hold on the Investigational New Drug (IND) application for fordadistrogene movaparvovec by the U.S. Food and Drug Administration (FDA).

In a statement released to the media on April 28, 2022, Pfizer said that it has addressed the agency’s requests related to the potency assay.

The global Phase 3 study has been ongoing in 11 countries and was paused in December 2021 to implement a protocol amendment following a fatal serious adverse event that occurred in a Phase 1b study in the non-ambulatory cohort, the company informed.

Pfizer said that it expects to restart the trials by the end of June 2022, in all the sites where the trial has been put on hold after receiving approvals from the regulatory authorities in respective countries.

Meanwhile, the regulatory authorities in the United Kingdom, Canada, Taiwan, Spain and Belgium have approved the re-start of the Phase 3 study, Pfizer informed.

“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, Rare Disease, Pfizer 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,” she added.

It may be recalled that the trial was put on hold in December 2021 after a non-ambulatory participant who was in a more advanced stage of the disease with underlying cardiac dysfunction died due to adverse effects during phase 1b of the trial.

Duchenne muscular dystrophy is a serious genetic disorder that affects males more than females and occurs 1 in every 3,500 to 5,000 live male births, where the affected person develops progressive muscle degeneration and weakness.