Safe­ty fears force Pfiz­er to change piv­otal DMD gene ther­a­py tri­al pro­to­col

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.

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