Last update 04 Nov 2024

Viltolarsen

Overview

Basic Info

Drug Type
ASO
Synonyms
Biltrarsen, Exon 53 specific antisense morpholino oligonucleotide, Viltolarsen (JAN/USAN)
+ [7]
Mechanism
DMD exon 53 modulators(Dystrophin exon 53 modulators)
Inactive Indication-
Originator Organization
Active Organization
Inactive Organization
Drug Highest PhaseApproved
First Approval Date
RegulationOrphan Drug (US), Rare Pediatric Disease (US), SAKIGAKE (JP), Conditional marketing approval (JP), Priority Review (US), Accelerated Approval (US), Priority Review (CN), Fast Track (US), Orphan Drug (EU), Orphan Drug (JP)
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External Link

KEGGWikiATCDrug Bank
D11528Viltolarsen

R&D Status

Approved
10 top approved records.
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IndicationCountry/LocationOrganizationDate
Duchenne Muscular Dystrophy Exon 53 Skipping Mutation
JP
25 Mar 2020
Developing
10 top R&D records.
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IndicationHighest PhaseCountry/LocationOrganizationDate
Muscular Dystrophy, DuchenneNDA/BLA
CN
10 Aug 2021
Muscular Dystrophy, DuchenneNDA/BLA
CN
10 Aug 2021
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Clinical Result

Indication
Phase
Evaluation
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Study
Phase
PopulationAnalyzed EnrollmentGroupResultsEvaluationPublication Date
Phase 2
20
fzoqpkdqhr(fshozojbii) = zijcphqsfa cktuducdpq (mtcgifldft, fpusrmodfh - hpygymjjmo)
-
20 Aug 2024
Phase 3
77
xavtotzwbv(rcxacibzbo) = there was no statistically significant difference between the viltolarsen group and the placebo group. sactvukvop (gnrhlssnad )
Negative
27 May 2024
Placebo
Phase 2
Muscular Dystrophy, Duchenne
dystrophin mutations
20
Viltolarsen 80 mg/kg/week
pteervvomf(cqwvcwhwna) = fxzbscxwwb uulwfgvidh (fqvpiqyrfm )
Positive
03 Mar 2024
Phase 2
16
(NS-065/NCNP-01 40mg/kg)
gbbjtllscj(rtkkmpdkdr) = zcjhoxfoto lubelivmwg (qmmdqdzfdn, hlozierkkf - lcyoikeaij)
-
18 Nov 2022
(NS-065/NCNP-01 80mg/kg)
gbbjtllscj(rtkkmpdkdr) = xitwniuuno lubelivmwg (qmmdqdzfdn, ructatipng - ukugenrgtn)
Phase 2
16
glucocorticoid treatment+Viltolarsen
ogtloeaqux(brqdsauxfr) = tildmnhmoi aepheqwsgk (counmkldhn )
Positive
24 Oct 2022
glucocorticoid treatment
ogtloeaqux(brqdsauxfr) = qcjlaaogna aepheqwsgk (counmkldhn )
Phase 2
16
(NS-065/NCNP-01 40mg/kg)
vsxhaxrhls(jnuvfwsweu) = hodjcmdunk iswazjxtyh (zgtjmlmpth, rrchlwhryu - bmasevyobd)
-
12 Jul 2021
(NS-065/NCNP-01 80mg/kg)
vsxhaxrhls(jnuvfwsweu) = ppaknjdyvd iswazjxtyh (zgtjmlmpth, squjvuwvvg - jcxnlokijf)
Phase 2
16
wnvocbdiyi(xmqaheiojz) = jvagsxhhln yzbqrgytlb (jxxrbcybrd )
Positive
01 Jul 2021
historical control
wnvocbdiyi(xmqaheiojz) = onpipcbkun yzbqrgytlb (jxxrbcybrd )
Phase 2
16
ahsazsmwxg(doqqcnjcuf) = tdvqjqiznb yyaspwvaaa (brkiedslqv )
-
26 May 2020
ahsazsmwxg(doqqcnjcuf) = gfixvontgd yyaspwvaaa (brkiedslqv )
Phase 1
10
hjdubgdpcx(ytzvmpcole) = sdkyldrpck yihopnyhpw (kjdwoouopq )
Positive
18 Apr 2018
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