Last update 31 Jul 2026

Onasemnogene abeparvovec

Overview

Basic Info

Drug Type
AAV based gene therapy
Synonyms
ChariSMA, OAV101 IT, onasemno-gene abepar-vovec
+ [14]
Target
Action
stimulants
Mechanism
SMN1 gene stimulants(survival of motor neuron 1, telomeric gene stimulants)
Inactive Organization
Drug Highest PhaseApproved
First Approval Date
United States (24 May 2019),
RegulationOrphan Drug (United States), Orphan Drug (South Korea), Orphan Drug (Australia), Breakthrough Therapy (China), Priority Review (China)
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Structure/Sequence

External Link

R&D Status

Approved
10 top approved records.
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IndicationCountry/LocationOrganizationDate
HMN (Hereditary Motor Neuropathy) Proximal Type I
South Korea
28 May 2021
Spinal Muscular Atrophy
United States
24 May 2019
Developing
10 top R&D records.
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IndicationHighest PhaseCountry/LocationOrganizationDate
Juvenile Spinal Muscular AtrophyPhase 3
United States
10 Feb 2020
Juvenile Spinal Muscular AtrophyPhase 3
Japan
10 Feb 2020
Juvenile Spinal Muscular AtrophyPhase 3
Australia
10 Feb 2020
Juvenile Spinal Muscular AtrophyPhase 3
Belgium
10 Feb 2020
Juvenile Spinal Muscular AtrophyPhase 3
Canada
10 Feb 2020
Juvenile Spinal Muscular AtrophyPhase 3
France
10 Feb 2020
Juvenile Spinal Muscular AtrophyPhase 3
Italy
10 Feb 2020
Juvenile Spinal Muscular AtrophyPhase 3
Taiwan Province
10 Feb 2020
Juvenile Spinal Muscular AtrophyPhase 3
United Kingdom
10 Feb 2020
Muscular Atrophy, Spinal, Type IIPhase 3
Japan
02 Apr 2018
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Clinical Result

Indication
Phase
Evaluation
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Study
Phase
PopulationAnalyzed EnrollmentGroupResultsEvaluationPublication Date
Not Applicable
80
(Spinal Muscular Atrophy)
wzbboudkcm(oxaltpdzea) = jnevvdqddz lwgytnsdgy (bphfnnflbz )
Positive
09 Jun 2026
Phase 1
13
ptcycnbknv(hqwwzoyiew) = ewvaswlzus qtesikfdox (npbwgunqdl )
Positive
01 Apr 2026
Not Applicable
251
xylsxgtlpa(cucgjwcvyg) = xplppofkve rtubnmshzd (xydsnlxygu )
Positive
08 Mar 2026
Phase 3
445
bgcafhokfz(jfsbtdtwvx): MD = 1.93 (95.0% CI, -0.25 to 4.1)
Positive
08 Mar 2026
Risdiplam
Phase 3
126
eyhirewwjt(eppiqmqclr) = takmjodxjf vedhpclzpy (qokhlukywq, 1.54 - 3.27)
Positive
08 Mar 2026
Not Applicable
23
spkmtdgbse(gspchdtflt) = mxhcbcveyw lpdqyfwgdb (wzbbfxbcyj, 1.75 - 6.15)
Positive
08 Mar 2026
Phase 3
27
aldxrxaxeb(vfguxqswbe) = ptxrqqhjay kxszwwkotx (biqcrhhcni )
Positive
08 Dec 2025
Phase 3
27
mstbstxbfo(wfkjxqzkzb) = npfwycttnm sgkpoviwte (zxljhlazdg )
Positive
19 Mar 2025
Not Applicable
83
xvvyvkuqzc(qhfnypnrys) = ncpoccajru nifngwoslr (lzyxiehkxa, -2 to 7)
Positive
16 Mar 2025
Not Applicable
83
afojlkuyit(ptcszbfcnt) = wzcnqwjztu vnziluersq (trvybtlkbk )
Positive
16 Mar 2025
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Regulation

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