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September 28, 2021 10:49 AM EDT
R&DCell/Gene Tx
Safety fears force Pfizer to change pivotal DMD gene therapy trial protocol
Kyle Blankenship
Managing Editor
As one of the biggest players in an increasingly packed gene therapy space, Pfizer has taken an early lead over specialists like Sarepta in taking a Duchenne muscular dystrophy (DMD) candidate into late-stage testing. But new safety fears have led Pfizer to scale back that trial, cutting out patients with certain genetic mutations.

Pfizer has amended its enrollment protocol for a Phase III test for gene therapy fordadistrogene movaparvovec in DMD after investigators flagged severe side effects tied to specific mutations, according to a letter the drugmaker sent to Parent Project Muscular Dystrophy, a patient advocacy group.

The amended CIFFREO study plans to enroll 99 DMD patients without any mutations affecting exons 9 through 13, or a deletion that affects both exon 29 and exon 30. Exons are portions of a gene that code for specific amino acids.