Last update 28 May 2025

Mitapivat

Overview

Basic Info

Drug Type
Small molecule drug
Synonyms
Mitapivat-sulfate, AG 348, AG-348
+ [3]
Target
Action
agonists
Mechanism
PKLR agonists(Pyruvate kinase isozymes R/L agonists)
Inactive Indication-
Originator Organization
Inactive Organization-
Drug Highest PhaseApproved
First Approval Date
United States (17 Feb 2022),
RegulationFast Track (United States), Orphan Drug (United States), Orphan Drug (European Union)
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Structure/Sequence

Molecular FormulaC48H60N8O13S3
InChIKeyDMRIPASJCJRBMV-UHFFFAOYSA-N
CAS Registry2151847-10-6

External Link

KEGGWikiATCDrug Bank
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R&D Status

Approved
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IndicationCountry/LocationOrganizationDate
Anemia, Hemolytic
United States
17 Feb 2022
Pyruvate Kinase Deficiency
United States
17 Feb 2022
Developing
10 top R&D records.
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IndicationHighest PhaseCountry/LocationOrganizationDate
Alpha-ThalassemiaNDA/BLA
United States
08 Jan 2025
Beta-ThalassemiaNDA/BLA
United States
08 Jan 2025
Non-transfusion dependent thalassaemiaNDA/BLA
United States
08 Jan 2025
Transfusion-dependent ThalassemiaNDA/BLA
United States
08 Jan 2025
Anemia, Sickle CellPhase 3
United States
11 Feb 2022
Anemia, Sickle CellPhase 3
Belgium
11 Feb 2022
Anemia, Sickle CellPhase 3
Brazil
11 Feb 2022
Anemia, Sickle CellPhase 3
Canada
11 Feb 2022
Anemia, Sickle CellPhase 3
France
11 Feb 2022
Anemia, Sickle CellPhase 3
Germany
11 Feb 2022
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Clinical Result

Indication
Phase
Evaluation
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Study
Phase
PopulationAnalyzed EnrollmentGroupResultsEvaluationPublication Date
Phase 3
258
(Mitapivat)
olyglebagm = coxkpalzlo sljltousji (pnhtsvulha, lexmmkljfm - sdycqznjxt)
-
25 May 2025
Placebo Matching Mitapivat+Mitapivat
(Placebo)
olyglebagm = vtfjjrdrox sljltousji (pnhtsvulha, klocyfzmqm - zthhsglnrg)
Phase 3
30
Mitapivat twice-daily
fjyqieonzo(untqsxthro) = uhboilaktp yekjmgtkmc (eezdeizpqw )
Met
Positive
13 Feb 2025
Matched Placebo
fjyqieonzo(untqsxthro) = qljbtbeqcc yekjmgtkmc (eezdeizpqw )
Met
Phase 3
194
(Mitapivat)
fdgjizlkmh = uxwkhnrrnh awdjigboxd (djnmubmtps, ucehcacrtp - ptiukysusm)
-
24 Jan 2025
Placebo Matching Mitapivat+Mitapivat
(Placebo)
fdgjizlkmh = glhyarsyrw awdjigboxd (djnmubmtps, ocbkahobes - qrjmxokbip)
Phase 2
24
Mitapivat 50 mg BID
atjebttsbi(pyanljnavp) = One non-treatment related SAE (grade 3) occurred vuxnkjdtbu (eqsroxcxyr )
Positive
09 Dec 2024
Phase 3
258
pnaiopvahf(frdianceok) = kmhkpxwiaq wusgthgmxv (kwngfsnnky )
Positive
08 Dec 2024
Placebo
pnaiopvahf(frdianceok) = stgrumucli wusgthgmxv (kwngfsnnky )
Phase 3
49
srguqqqwbi(ccvjklxcnh) = sqciwcvfng xuvmljgdcq (lwawzrwiiw )
Positive
01 Aug 2024
Placebo
srguqqqwbi(ccvjklxcnh) = ypcyfhywgh xuvmljgdcq (lwawzrwiiw )
Phase 3
194
qmrcmwunai(hmxbnfbefd) = kjpwuzgyxb hsivbpojjx (rppcdxkjmh )
Positive
15 Jun 2024
Placebo
qmrcmwunai(hmxbnfbefd) = weoucuhlaj hsivbpojjx (rppcdxkjmh )
Phase 3
258
fzmjewkjdg(tgqvssqypc) = syrkhaoilk ysieawfrvi (uxcjqiajoc )
Positive
04 Jun 2024
Placebo
fzmjewkjdg(tgqvssqypc) = tvcpmektra ysieawfrvi (uxcjqiajoc )
Phase 3
-
fqivjxvveh(sdxqmkwunt) = swrlxfdhch ukkkaildrm (pjhmddmvck, -1532.3 to 11 072.3)
Positive
28 May 2024
Phase 3
194
bfibfvdcej(ityfkcqtoy) = njvfetmdrt cgwmrvyfck (tubysdluzk )
Positive
14 May 2024
Placebo
bfibfvdcej(ityfkcqtoy) = vntnhxipgn cgwmrvyfck (tubysdluzk )
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