Date:
October 19, 2020
Issue #:
1609
Summary:
The antisense oligonucleotide viltolarsen (Viltepso –
NS Pharma) 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 53 skipping
(DMD-53). It is the second drug to be approved for this
indication; the antisense oligonucleotide golodirsen
(Vyondys 53) was approved in 2019.
NS Pharma) 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 53 skipping
(DMD-53). It is the second drug to be approved for this
indication; the antisense oligonucleotide golodirsen
(Vyondys 53) was approved in 2019.
