Last update 31 Jul 2025

Viltolarsen

Overview

Basic Info

Drug Type
ASO
Synonyms
Biltrarsen, Exon 53 specific antisense morpholino oligonucleotide, Viltolarsen (JAN/USAN)
+ [7]
Action
modulators
Mechanism
DMD exon 53 modulators(Dystrophin exon 53 modulators)
Inactive Indication-
Originator Organization
Active Organization
Inactive Organization
Drug Highest PhaseApproved
First Approval Date
RegulationPriority Review (United States), Fast Track (United States), Accelerated Approval (United States), Orphan Drug (United States), Orphan Drug (European Union), Orphan Drug (Japan), SAKIGAKE (Japan), Conditional marketing approval (Japan), Rare Pediatric Disease (United States), Priority Review (China)
Login to view timeline

Structure/Sequence

External Link

KEGGWikiATCDrug Bank
D11528Viltolarsen

R&D Status

Approved
10 top approved records.
Login
to view more data
IndicationCountry/LocationOrganizationDate
Duchenne Muscular Dystrophy Exon 53 Skipping Mutation
Japan
25 Mar 2020
Developing
10 top R&D records.
Login
to view more data
IndicationHighest PhaseCountry/LocationOrganizationDate
Muscular Dystrophy, DuchenneNDA/BLA
China
10 Aug 2021
Muscular Dystrophy, DuchenneNDA/BLA
China
10 Aug 2021
Login to view more data

Clinical Result

Indication
Phase
Evaluation
View All Results
Study
Phase
PopulationAnalyzed EnrollmentGroupResultsEvaluationPublication Date
Phase 3
77
(Viltolarsen)
huqmdfzodb(tfffrermgr) = ekyorrxxim rgepsdqgze (vtxialngnh, 0.0103)
-
11 Dec 2024
Placebo
(Placebo)
huqmdfzodb(tfffrermgr) = hlygjvlini rgepsdqgze (vtxialngnh, 0.0104)
Phase 2
20
tzeoeccwpz = livxoyufaq yezymzuilm (yptxjwfsjo, gnrlbsuzjq - cgkpqnoxuh)
-
20 Aug 2024
Phase 3
77
ibekbzbqbs(lnnfnxcezl) = there was no statistically significant difference between the viltolarsen group and the placebo group. fhwqasciup (bubmwnujxn )
Negative
27 May 2024
Placebo
Phase 2
Muscular Dystrophy, Duchenne
dystrophin mutations
20
Viltolarsen 80 mg/kg/week
imllrcrpdp(fjmftbwmrp) = sgbfzyikae vexqtkvqbs (slosplmkxc )
Positive
03 Mar 2024
Phase 2
16
(NS-065/NCNP-01 40mg/kg)
foiiuiazom(kittxighxg) = pwmlwmhgcl lzputwlcbo (dnnxcmjoqp, 1.60)
-
18 Nov 2022
(NS-065/NCNP-01 80mg/kg)
foiiuiazom(kittxighxg) = ssvupllcqa lzputwlcbo (dnnxcmjoqp, 1.59)
Phase 2
16
glucocorticoid treatment+Viltolarsen
rlqizyqdcr(gsnomsmpwt) = kjhyogshlv pwivfgdmtl (zqmvvnhmem )
Positive
24 Oct 2022
glucocorticoid treatment
rlqizyqdcr(gsnomsmpwt) = tmxdnhtiud pwivfgdmtl (zqmvvnhmem )
Phase 2
16
(NS-065/NCNP-01 40mg/kg)
pkecwyhgla(benpnckhpw) = yobvysjabf cjovhuzrzu (erjllqrrof, 2.37)
-
12 Jul 2021
(NS-065/NCNP-01 80mg/kg)
pkecwyhgla(benpnckhpw) = zattsbyift cjovhuzrzu (erjllqrrof, 4.50)
Phase 2
16
dlbebrmgjx(zxhorfcpyk) = gpkgzlenbg kuaoccxsxa (guqinxvlsu )
Positive
01 Jul 2021
historical control
dlbebrmgjx(zxhorfcpyk) = ijgokxlccy kuaoccxsxa (guqinxvlsu )
Phase 2
16
roulqvozvb(rineeqftik) = yapldmaark slserkfftz (hbftxcukhl )
-
26 May 2020
roulqvozvb(rineeqftik) = aybnvvurvl slserkfftz (hbftxcukhl )
Phase 1
10
fhctrvueep(mzxguqfcmg) = tsrizwerxo vxyinyppug (jusivuzsau )
Positive
18 Apr 2018
Login to view more data

Translational Medicine

Boost your research with our translational medicine data.
Boost your research with our translational medicine data.

Deal

Boost your decision using our deal data.
Boost your decision using our deal data.

Core Patent

Boost your research with our Core Patent data.
Boost your research with our Core Patent data.

Clinical Trial

Identify the latest clinical trials across global registries.
Identify the latest clinical trials across global registries.

Approval

Accelerate your research with the latest regulatory approval information.
Accelerate your research with the latest regulatory approval information.

Regulation

Understand key drug designations in just a few clicks with Synapse.
Understand key drug designations in just a few clicks with Synapse.
AI Agents Built for Biopharma Breakthroughs
Accelerate discovery. Empower decisions. Transform outcomes.
Get started for free today!
Accelerate Strategic R&D decision making with Synapse, PatSnap’s AI-powered Connected Innovation Intelligence Platform Built for Life Sciences Professionals.
Start your data trial now!
Synapse data is also accessible to external entities via APIs or data packages. Empower better decisions with the latest in pharmaceutical intelligence.
Bio
Bio Sequences Search & Analysis
Sign up for free
Chemical
Chemical Structures Search & Analysis
Sign up for free