Last update 01 Nov 2024

Sepofarsen

Overview

Basic Info

Drug Type
ASO
Synonyms
QR-110
Target
Mechanism
Cep290 modulators(Centrosomal protein of 290 kDa modulators), Gene expression stimulants
Originator Organization
Active Organization
Inactive Organization-
Drug Highest PhasePhase 2/3
First Approval Date-
RegulationOrphan Drug (US), Rare Pediatric Disease (US), PRIME (EU), Fast Track (US), Orphan Drug (EU)
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External Link

KEGGWikiATCDrug Bank
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R&D Status

10 top R&D records.
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IndicationHighest PhaseCountry/LocationOrganizationDate
Retinal DystrophiesPhase 3
BE
23 Mar 2021
Retinal DystrophiesPhase 3
BR
23 Mar 2021
Retinal DystrophiesPhase 3
CA
23 Mar 2021
Retinal DystrophiesPhase 3
DE
23 Mar 2021
Retinal DystrophiesPhase 3
IT
23 Mar 2021
Retinal DystrophiesPhase 3
NL
23 Mar 2021
Retinal DystrophiesPhase 3
GB
23 Mar 2021
BlindnessPhase 3
US
04 Apr 2019
BlindnessPhase 3
BE
04 Apr 2019
BlindnessPhase 3
BR
04 Apr 2019
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Clinical Result

Indication
Phase
Evaluation
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Study
Phase
PopulationAnalyzed EnrollmentGroupResultsEvaluationPublication Date
Not Applicable
-
-
neyalelybd(zgdlaqbdkr) = tldcggnbxd ipqnswmbwv (vabzpwuudz )
-
23 Apr 2023
neyalelybd(zgdlaqbdkr) = vamtgjvnfl ipqnswmbwv (vabzpwuudz )
Phase 1/2
9
sxqchxlcgh(fywkgsbaqd) = dswrhtihop hljssmmnog (khgtneglux, zkhxqpuggd - rnaqakixxf)
-
04 Jan 2023
Phase 1/2
11
(Untreated eye)
eacgshunvo(ipvxajkqde) = Eight patients developed cataracts, of which six (75.0%) were categorized as serious (2/3 with 160 µg/80 µg; 4/5 with 320 µg/160 µg), as lens replacement was required. pztiytzjrh (souviorird )
Positive
04 Apr 2022
Phase 1/2
9
evepjafvqy(rofjwlmwjz) = One patient developed cataract at Month 9 in the second treated eye wpjkwhvjho (zcyvjosxze )
-
01 Jun 2021
Phase 1/2
Leber Congenital Amaurosis 10
p.Cys998X mutation in the CEP290 gene
11
Sepofarsen 320/160-µg
aoctidjxbd(zqhjuefzij) = Reported cases of cataract were 3 in the 160/80-µg group and 5 in the 320/160-µg group crcoqeudut (fuzenrghog )
Positive
13 Nov 2020
Phase 1/2
11
cmywuygzxd(qsabilwhjp) = Although sepofarsen treatment is associated with cataract development, it is well tolerated and shows improvement in BCVA, FST, and mobility course. The 160/80 µg dose has a more favorable benefit:risk profile than 320/160 µg bhylitiuta (cfrnqwayzv )
-
01 Jun 2020
Phase 1/2
11
vrmsgdfbos(ekbjqxnexi) = xucrwvnarx bubynrbxsq (qwuepoyneq )
Positive
10 Oct 2019
Placebo
vrmsgdfbos(ekbjqxnexi) = ikwopcxayk bubynrbxsq (qwuepoyneq )
Phase 1/2
10
zzqjawoghr(mtjduhipof) = results also demonstrated greater stability asuujsbowg (vlbxuordgv )
Positive
01 Jul 2019
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