Date:
July 27, 2020
Issue #:
1603
Summary:
Golodirsen (Vyondys 53 — Sarepta), an antisense
oligonucleotide, has received accelerated approval
from the FDA for treatment of Duchenne muscular
dystrophy (DMD) in the ~8% of patients who have
mutations of the dystrophin gene that are amenable
to exon 53 skipping. It is the first drug to be approved
for this indication and the third to be approved for
treatment of DMD; the antisense oligonucleotide
eteplirsen (Exondys 51) and the oral corticosteroid
deflazacort (Emflaza) were approved earlier.
oligonucleotide, has received accelerated approval
from the FDA for treatment of Duchenne muscular
dystrophy (DMD) in the ~8% of patients who have
mutations of the dystrophin gene that are amenable
to exon 53 skipping. It is the first drug to be approved
for this indication and the third to be approved for
treatment of DMD; the antisense oligonucleotide
eteplirsen (Exondys 51) and the oral corticosteroid
deflazacort (Emflaza) were approved earlier.
