Date:
June 28, 2021
Issue #:
1627
Summary:
Casimersen (Amondys 45 – Sarepta), an IV antisense
oligonucleotide, has received accelerated approval
from the FDA for treatment of Duchenne muscular
dystrophy (DMD) in patients who have mutations
of the dystrophin gene that are amenable to exon
45 skipping (DMD-45), which occur in ~8% of DMD
cases. Casimersen is the first drug to be approved
for this indication and the fifth to be approved for
treatment of DMD; the IV antisense oligonucleotides
eteplirsen (Exondys 51), golodirsen (Vyondys 53),
and viltolarsen (Viltepso) and the oral corticosteroid
deflazacort (Emflaza) were approved earlier. The
DMD gene consists of 79 exons; its long length makes
it susceptible to mutations and deletions.
oligonucleotide, has received accelerated approval
from the FDA for treatment of Duchenne muscular
dystrophy (DMD) in patients who have mutations
of the dystrophin gene that are amenable to exon
45 skipping (DMD-45), which occur in ~8% of DMD
cases. Casimersen is the first drug to be approved
for this indication and the fifth to be approved for
treatment of DMD; the IV antisense oligonucleotides
eteplirsen (Exondys 51), golodirsen (Vyondys 53),
and viltolarsen (Viltepso) and the oral corticosteroid
deflazacort (Emflaza) were approved earlier. The
DMD gene consists of 79 exons; its long length makes
it susceptible to mutations and deletions.
