Casimersen (Amondys 45) for Duchenne Muscular Dystrophy (online only)

Date: June 28, 2021
Issue #: 
1627Summary: 
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 fi rst 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.

Source: The Medical Letter - June 15, 2021 Category: Drugs & Pharmacology Authors: admin Tags: Amondys 45 casimersen deflazacort Duchenne muscular dystrophy Emflaza eteplirsen Exondys 51 golodirsen Viltepso viltolarsen Vyondys 53 Source Type: research