Drug Profile

Pacritinib - CTI BioPharma

Alternative Names: ENPAXIQ; Epjevy; ONX-0803; Pacritinib citrate - CTI BioPharma; Pacritinibum; SB-1518; VONJO

Latest Information Update: 02 Jun 2025

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At a glance

Originator S*BIO
Developer Cardiff University; CTI BioPharma; National Cancer Research Institute; S*BIO
Class Antineoplastics; Aza compounds; Benzene derivatives; Ethers; Macrocyclic compounds; Polycyclic bridged compounds; Pyrimidines; Pyrrolidines; Small molecules
Mechanism of Action Fms-like tyrosine kinase 3 inhibitors; Interleukin-1 receptor-associated kinase inhibitors; Janus kinase 2 inhibitors; Macrophage colony-stimulating factor receptor antagonists

Orphan Drug Status

Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

Yes - Myelofibrosis
New Molecular Entity Yes

Marketed Myelofibrosis
Phase II Graft-versus-host disease; VEXAS syndrome
Discontinued Acute myeloid leukaemia; Chronic myeloid leukaemia; Colorectal cancer; Haematological disorders; Lymphoma; Myelodysplastic syndromes; Myeloproliferative disorders; SARS-CoV-2 acute respiratory disease

12 May 2025 Phase-II clinical trials in VEXAS syndrome (Treatment-experienced) in Spain (PO) (NCT06782373)
17 Jan 2025 Swedish Orphan Biovitrum plans a phase II trial for VEXAS Syndrome in USA, Canada, France, Germany, Italy, Spain and United Kingdom, in May 2025 (PO, Capsules) (NCT06782373, EUCT2024-516347-41-00, PAC601)
17 Dec 2024 Pacritinib is still in phase-III clinical trials in Myelofibrosis in Belarus, Serbia, Georgia, Ukraine and Israel (NCT03165734)

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