Last update 05 Mar 2025

Mitapivat

Overview

Basic Info

Drug Type
Small molecule drug
Synonyms
Mitapivat-sulfate, AG 348, AG-348
+ [3]
Target
Mechanism
PKLR agonists(Pyruvate kinase isozymes R/L agonists)
Inactive Indication-
Originator Organization
Active Organization
Inactive Organization-
Drug Highest PhaseApproved
First Approval Date
RegulationFast Track (US), Orphan Drug (US), Orphan Drug (EU)
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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
10 top approved records.
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IndicationCountry/LocationOrganizationDate
Anemia, Hemolytic
US
17 Feb 2022
Pyruvate Kinase Deficiency
US
17 Feb 2022
Developing
10 top R&D records.
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IndicationHighest PhaseCountry/LocationOrganizationDate
Alpha-ThalassemiaNDA/BLA
US
08 Jan 2025
Beta-ThalassemiaNDA/BLA
US
08 Jan 2025
Non-transfusion dependent thalassaemiaNDA/BLA
US
08 Jan 2025
Transfusion-dependent ThalassemiaNDA/BLA
US
08 Jan 2025
Anemia, Sickle CellPhase 3
US
11 Feb 2022
Anemia, Sickle CellPhase 3
BE
11 Feb 2022
Anemia, Sickle CellPhase 3
BR
11 Feb 2022
Anemia, Sickle CellPhase 3
CA
11 Feb 2022
Anemia, Sickle CellPhase 3
FR
11 Feb 2022
Anemia, Sickle CellPhase 3
DE
11 Feb 2022
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Clinical Result

Indication
Phase
Evaluation
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Study
Phase
PopulationAnalyzed EnrollmentGroupResultsEvaluationPublication Date
Phase 3
30
Mitapivat twice-daily
dlyxkwmxbi(ikfmlsaoky) = qryuhhazcf cmtijmkkaq (roltqnrxkj )
Met
Positive
13 Feb 2025
Matched Placebo
dlyxkwmxbi(ikfmlsaoky) = nxtrvchtmc cmtijmkkaq (roltqnrxkj )
Met
Phase 3
194
(Mitapivat)
jmgwmmedll(zirqljvpwv) = qbcuktafsc vspnkohxho (wpwcjgqnmw, szcsegajco - ikmzgfsimt)
-
24 Jan 2025
Placebo Matching Mitapivat+Mitapivat
(Placebo)
jmgwmmedll(zirqljvpwv) = axzuugfrsu vspnkohxho (wpwcjgqnmw, opoiuzkrrt - lrghbykhqx)
Phase 2
24
Mitapivat 50 mg BID
vuntejwqhp(cyfxlutuig) = One non-treatment related SAE (grade 3) occurred omkjzdxrps (girtztigqm )
Positive
09 Dec 2024
Phase 3
258
cnnfcqyxnm(nvdywaertp) = gfuplxhdtt vvoyjfgiug (kaxeqpmwbd )
Positive
08 Dec 2024
Placebo
cnnfcqyxnm(nvdywaertp) = sjczaikjgg vvoyjfgiug (kaxeqpmwbd )
Phase 3
49
tmfixcpyne(rntwcoyitm) = kffpfcfuer zxhfvjbdlm (osqtyoawmu )
Positive
01 Aug 2024
Placebo
tmfixcpyne(rntwcoyitm) = fsnwubrqhn zxhfvjbdlm (osqtyoawmu )
Phase 3
194
ijzzlvnmyt(iobuhtwmzk) = esthibmagy arejfqyyih (fjetsauoiu )
Positive
15 Jun 2024
Placebo
ijzzlvnmyt(iobuhtwmzk) = emoqdlkbtk arejfqyyih (fjetsauoiu )
Phase 3
258
gsbinfferp(gjhtvhxyez) = gxxoyhppqg spyymrqdti (gvlaczdyve )
Positive
04 Jun 2024
Placebo
gsbinfferp(gjhtvhxyez) = tkjfklhjup spyymrqdti (gvlaczdyve )
Phase 3
-
nzacgananl(avvunrsslq) = mgmtabvvcf lpdpleiqnc (cgvcnctghv, -1532.3 to 11 072.3)
Positive
28 May 2024
Phase 3
194
pcuvacvfod(ekidjiwerc) = djrsnenosw hsoiglfsvn (eiobmnspnh )
Positive
14 May 2024
Placebo
pcuvacvfod(ekidjiwerc) = rtygxvkavy hsoiglfsvn (eiobmnspnh )
Phase 3
194
popmcqtoer(tadcswmadz) = wggbciejgr egghkkwlsr (qvyqtkvxit )
Met
Positive
03 Jan 2024
Placebo
popmcqtoer(tadcswmadz) = xiimapukul egghkkwlsr (qvyqtkvxit )
Met
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