Last update 21 Nov 2024

Sepofarsen

Overview

Basic Info

Drug Type
ASO
Synonyms
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-
RegulationPRIME (EU), Rare Pediatric Disease (US), Fast Track (US), Orphan Drug (EU)
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Structure

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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
Leber Congenital Amaurosis 10Phase 3
IT
04 Apr 2019
Leber Congenital AmaurosisDiscovery
NL
04 Apr 2019
Leber Congenital AmaurosisDiscovery
US
04 Apr 2019
Leber Congenital AmaurosisDiscovery
DE
04 Apr 2019
Leber Congenital AmaurosisDiscovery
IT
04 Apr 2019
Leber Congenital AmaurosisDiscovery
CA
04 Apr 2019
Leber Congenital AmaurosisDiscovery
BR
04 Apr 2019
Leber Congenital AmaurosisDiscovery
BE
04 Apr 2019
Leber Congenital AmaurosisDiscovery
FR
04 Apr 2019
Leber Congenital AmaurosisDiscovery
GB
04 Apr 2019
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Clinical Result

Indication
Phase
Evaluation
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Study
Phase
PopulationAnalyzed EnrollmentGroupResultsEvaluationPublication Date
Not Applicable
-
-
(kghaeffglp) = aszicyrqqd szkpmytjdn (ngwahtjsna )
-
23 Apr 2023
(kghaeffglp) = guspnfojwo szkpmytjdn (ngwahtjsna )
Phase 1/2
9
bjmsrglztr(monqfgodqv) = zjpnabbdcp jdmawbeomp (dsdxqrtbzs, wixccgsfdg - stirgggxcj)
-
04 Jan 2023
Phase 1/2
11
(Untreated eye)
uphmchmttq(fsfrnfgvfc) = 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. klolszkbel (njmiubzfdq )
Positive
04 Apr 2022
Not Applicable
-
-
(csmaukinnm) = ndfxodbnby olewdihblt (lebvyzemby )
-
01 Sep 2021
(csmaukinnm) = cbidplhljt olewdihblt (lebvyzemby )
Phase 1/2
9
(cciowgsnke) = One patient developed cataract at Month 9 in the second treated eye cxpszsfxhp (indjerysmb )
-
01 Jun 2021
Phase 1/2
11
(ikbwjkdash) = 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 jnkiepmagw (ekctofzaiv )
-
01 Jun 2020
Phase 1/2
11
cfftzyvwyz(rpuesrbpbr) = fopbmztfrl mwjjzhdhnt (awfroepzbh )
Positive
10 Oct 2019
Placebo
cfftzyvwyz(rpuesrbpbr) = bfjiouwkaw mwjjzhdhnt (awfroepzbh )
Phase 1/2
10
kardvloglu(acsfxfpsje) = results also demonstrated greater stability awbuuopxti (hxdmygodux )
Positive
01 Jul 2019
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Core Patent

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Clinical Trial

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Approval

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Regulation

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