|Trade names||Vonjo, Enpaxiq|
|Side effects||Diarrhea, low platelets, nausea, low hemoglobin, swollen legs|
|Typical dose||200 mg BID|
|Chemical and physical data|
|Molar mass||472.589 g·mol−1|
|3D model (JSmol)|
Pacritinib, sold under the brand name Vonjo, is an medication used to treat myelofibrosis. Specifically it is used in those with a platelet count less than 50 × 109/L in who it decreases the size of the spleen. It is taken by mouth.
Common side effects include diarrhea, low platelets, nausea, low hemoglobin, and swollen legs. Other side effects may include bleeding, prolonged QT, deep vein thrombosis, cancer, and infection. Use is not recommended in those with significant liver or kidney problems. It is a kinase inhibitor that blocks the action of Janus kinase 2 (JAK2) and fms-like tyrosine kinase 3\CD135 (FLT3).
Pacritinib was approved for medical use in the United States in 2022. Its application for approval in Europe was withdrawn in 2017 due to concerns it worsened outcomes. In the United States a month of medication costs about 23,000 USD as of 2022.
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.
It is taken at a dose of 200 mg twice per day.
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. Effectiveness was determined based upon the proportion of participants who had a 35% or greater spleen volume reduction from baseline to week 24. 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.
Society and culture
- "Vonjo- pacritinib capsule". DailyMed. 7 March 2022. Archived from the original on 24 March 2022. Retrieved 24 March 2022.
- "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. This article incorporates text from this source, which is in the public domain.
- "Enpaxiq: Withdrawal of the marketing authorisation application". Archived from the original on 4 August 2020. Retrieved 11 December 2022.
- "Vonjo Prices, Coupons, Copay & Patient Assistance". Drugs.com. Retrieved 11 December 2022.
- 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.
- World Health Organization (2011). "International nonproprietary names for pharmaceutical substances (INN): recommended INN: list 66". WHO Drug Information. 25 (3). hdl:10665/74683.