Drugdocs | Viltolarsen

Drug updated on 3/28/2024

Dosage Form	Injection (intravenous: 250 mg/5 mL (50 mg/mL))
Drug Class	Antisense oligonucleotides
Ongoing and Completed Studies	ClinicalTrials.gov

Indication

For the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 53 skipping.

Viltolarsen (Viltepso) is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 53 skipping.
One randomized controlled trial document was reviewed, providing information on safety, tolerability and efficacy of viltolarsen from a phase 2 study.
The study involved participants aged between 4 to 9 years with DMD amenable to exon 53 skipping; all were male and majority were white with an average age of approximately seven years old.
Participants received either low dose or high dose viltolarsen administered by weekly intravenous infusion over a period ranging from twenty to twenty-four weeks resulting in significant drug-induced dystrophin production across both dosage groups.
Compared against natural history controls, all sixteen participants treated with viltolarsen showed significant improvements in timed function tests including time taken to stand up from supine position, time taken run/walk ten meters and distance covered during six-minute walk test at week twenty-five visit post-treatment initiation.
Viltolarsen was well tolerated among the participant group; no adverse events required any reduction or discontinuation of dosage nor did any serious adverse events or deaths occur during this particular clinical trial study period indicating its relative safety compared to other treatments available for DMD patients within this demographic cohort.

Document Title	Year	Source
Viltepso (viltolarsen) Prescribing Information.	2021	NS Pharma, Inc., Paramus, NJ

Document Title	Sex Distribution	Year	Source
Safety, Tolerability, and Efficacy of Viltolarsen in Boys With Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping: A Phase 2 Randomized Clinical Trial.	16Subjects F: 0% M: 100%	2020	JAMA Neurol

Document Title

Sex Distribution:

F:0%

M:100%

16Subjects

Year:

2020

Source:JAMA Neurol