What is the role of the biologic agents eteplirsen, golodirsen, and viltolarsen in the management of muscular dystrophy?

Updated: Aug 17, 2020
  • Author: Twee T Do, MD; Chief Editor: Jeffrey D Thomson, MD  more...
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An open-label, phase 2, dose-escalation study evaluated the safety and efficacy of intravenously administered eteplirsen phosphorodiamidate morpholino oligomer (PMO) in patients with Duchenne MD. [37] Using data from 19 ambulant patients aged 5-15 years with amenable deletions in Duchenne MD, the investigators noted that eteplirsen was well tolerated with no serious drug-related adverse events; eteplirsen induced exon 51 skipping in all cohorts and new dystrophin protein expression in a significant dose-dependent, but variable, manner in boys (dose 2 mg/kg) onward.

Early clinical results of eteplirsen were biochemically promising for dystrophin production without significant adverse effects; however, functional ambulatory changes were not as consistently correlated. [37] Reevaluation of the existing data led to approval of eteplirsen in September 2016.


Golodirsen, a second antisense oligonucleotide, was approved by the FDA in December 2019. Golodirsen is indicated for treatment of Duchenne MD in patients who have a confirmed mutation of the DMD gene that is amenable to exon 53 skipping. Approval was based on an increase in dystrophin production in skeletal muscle observed in patients who were treated. Mean dystrophin levels increased from 0.1% of normal at baseline to 1.02% of normal by week 48, with a mean change in dystrophin of 0.92% of normal levels; the median change from baseline was 0.88%. [38]


Viltolarsen, another morpholino antisense oligomer, was approved by the FDA in August 2020 for Duchenne MD in patients with genetic mutations that are amenable to exon 53 skipping. Approval was based on results from a phase 2 two-period study in patients aged 4 to less than 10 years conducted in North America (N = 16) and a multicenter, open-label study in boys aged 5 to less than 18 years conducted in Japan (N = 16). In the former, 100% of patients who received the recommended dose of 80 mg/kg/wk (N = 8) showed an increase in dystrophin levels after treatment. [39]

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