Etavopivat

Etavopivat
Clinical data
Other namesFT-4202; NN 7535; NN-7536; NN7535
Legal status
Legal status
  • investigational
Identifiers
  • (2S)-1-[5-(2,3-dihydro-[1,4]dioxino[2,3-b]pyridin-7-ylsulfonyl)-1,3,4,6-tetrahydropyrrolo[3,4-c]pyrrol-2-yl]-3-hydroxy-2-phenylpropan-1-one
CAS Number
PubChem CID
IUPHAR/BPS
DrugBank
ChemSpider
UNII
KEGG
ChEBI
ChEMBL
Chemical and physical data
FormulaC22H23N3O6S
Molar mass457.50 g·mol−1
3D model (JSmol)
  • C1COC2=C(O1)C=C(C=N2)S(=O)(=O)N3CC4=C(C3)CN(C4)C(=O)[C@H](CO)C5=CC=CC=C5
  • InChI=InChI=1S/C22H23N3O6S/c26-14-19(15-4-2-1-3-5-15)22(27)24-10-16-12-25(13-17(16)11-24)32(28,29)18-8-20-21(23-9-18)31-7-6-30-20/h1-5,8-9,19,26H,6-7,10-14H2/t19-/m1/s1
  • Key:KZFFYEPYCVDOGE-LJQANCHMSA-N

Etavopivat is an investigational new drug that is being evaluated by FORMA Therapeutics for the treatment of sickle cell disease and thalassemia. It is an allosteric activator of the erythrocyte pyruvate kinase R (PKR) enzyme.[1][2][3]

Clinical trials

Etavopivat is in phase 3 clinical trials for sickle cell disease and thalassemia.[4]

References

  1. ↑ "Etavopivat - FORMA Therapeutics". AdisInsight. Springer Nature Switzerland AG.
  2. ↑ Matte A, Federti E, De Franceschi L (May 2023). "Erythrocyte pyruvate kinase activation in red cell disorders". Current Opinion in Hematology. 30 (3): 93–98. doi:10.1097/MOH.0000000000000758. PMID 36853806.
  3. ↑ Musallam KM, Andemariam B (June 2026). "Pyruvate Kinase Activation in the Management of Thalassemia and Sickle Cell Disease". Hemoglobin: 1–13. doi:10.1080/03630269.2026.2682386. PMID 42261266.
  4. ↑ Clinical trial number NCT06609226 for "A Research Study Looking at Long-term Treatment With Etavopivat in People With Sickle Cell Disease or Thalassaemia" at ClinicalTrials.gov