Pacritinib

Pacritinib
Names
Trade namesVonjo, Enpaxiq
Other namesSB1518
IUPAC name
  • (16E)-11-[2-(1-Pyrrolidinyl)ethoxy]-14,19-dioxa-5,7,26-triazatetracyclo[19.3.1.12,6.18,12]heptacosa-1(25),2(26),3,5,8,10,12(27),16,21,23-decaene
Clinical data
Main usesMyelofibrosis[1]
Side effectsDiarrhea, low platelets, nausea, low hemoglobin, swollen legs[1]
WHO AWaReUnlinkedWikibase error: ⧼unlinkedwikibase-error-statements-entity-not-set⧽
Routes of
use		By mouth
Typical dose200 mg BID[1]
External links
AHFS/Drugs.comMonograph
Legal
License data
Legal status
Chemical and physical data
FormulaC28H32N4O3
Molar mass472.589 g·mol−1
3D model (JSmol)
SMILES
  • c1cc2cc(c1)-c3ccnc(n3)Nc4ccc(c(c4)COC/C=C/COC2)OCCN5CCCC5
InChI
  • InChI=1S/C28H32N4O3/c1-2-13-32(12-1)14-17-35-27-9-8-25-19-24(27)21-34-16-4-3-15-33-20-22-6-5-7-23(18-22)26-10-11-29-28(30-25)31-26/h3-11,18-19H,1-2,12-17,20-21H2,(H,29,30,31)/b4-3+
  • Key:HWXVIOGONBBTBY-ONEGZZNKSA-N

Pacritinib, sold under the brand name Vonjo, is an medication used to treat myelofibrosis.[1] Specifically it is used in those with a platelet count less than 50 × 109/L in who it decreases the size of the spleen.[1] It is taken by mouth.[1]

Common side effects include diarrhea, low platelets, nausea, low hemoglobin, and swollen legs.[1] Other side effects may include bleeding, prolonged QT, deep vein thrombosis, cancer, and infection.[1] Use is not recommended in those with significant liver or kidney problems.[1] It is a kinase inhibitor that blocks the action of Janus kinase 2 (JAK2) and fms-like tyrosine kinase 3\CD135 (FLT3).[3]

Pacritinib was approved for medical use in the United States in 2022.[1] Its application for approval in Europe was withdrawn in 2017 due to concerns it worsened outcomes.[3] In the United States a month of medication costs about 23,000 USD as of 2022.[4]

Medical uses

Pacritinib in indicated to treat adults who have a rare form of a bone marrow disorder known as intermediate or high-risk primary or secondary myelofibrosis and who have platelet (blood clotting cells) levels below 50,000/µL.[1][2]

Dosage

It is taken at a dose of 200 mg twice per day.[1]

History

The effectiveness and safety of pacritinib were demonstrated in a study that included 63 participants with intermediate or high-risk primary or secondary myelofibrosis and low platelets who received pacritinib 200 mg twice daily or standard treatment.[2] Effectiveness was determined based upon the proportion of participants who had a 35% or greater spleen volume reduction from baseline to week 24.[2] Nine participants (29%) in the pacritinib treatment group had a 35% or greater spleen volume reduction, compared to one participant (3%) in the standard treatment group.[2]

The U.S. Food and Drug Administration (FDA) granted the application for pacritinib priority review, fast track, and orphan drug designations.[2]

Society and culture

Names

Pacritinib is the International nonproprietary name (INN).[5][6]

References

  1. 1 2 3 4 5 6 7 8 9 10 11 12 13 "Vonjo- pacritinib capsule". DailyMed. 7 March 2022. Archived from the original on 24 March 2022. Retrieved 24 March 2022.
  2. 1 2 3 4 5 6 "FDA approves drug for adults with rare form of bone marrow disorder". U.S. Food and Drug Administration. 1 March 2022. Archived from the original on 3 March 2022. Retrieved 3 March 2022. Public Domain This article incorporates text from this source, which is in the public domain.
  3. 1 2 "Enpaxiq: Withdrawal of the marketing authorisation application". Archived from the original on 4 August 2020. Retrieved 11 December 2022.
  4. ↑ "Vonjo Prices, Coupons, Copay & Patient Assistance". Drugs.com. Retrieved 11 December 2022.
  5. ↑ World Health Organization (2010). "International nonproprietary names for pharmaceutical substances (INN). proposed INN: list 104" (PDF). WHO Drug Information. 24 (4): 386. hdl:10665/74579. Archived (PDF) from the original on 2020-07-15. Retrieved 2022-10-13.
  6. ↑ World Health Organization (2011). "International nonproprietary names for pharmaceutical substances (INN): recommended INN: list 66". WHO Drug Information. 25 (3). hdl:10665/74683.
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