Pacritinib, sold under the brand name Vonjo, is an
anti-cancer medication
Chemotherapy (often abbreviated to chemo and sometimes CTX or CTx) is a type of cancer treatment that uses one or more anti-cancer drugs (chemotherapeutic agents or alkylating agents) as part of a standardized chemotherapy regimen. Chemotherap ...
used to treat
myelofibrosis
Primary myelofibrosis (PMF) is a rare bone marrow blood cancer. It is classified by the World Health Organization (WHO) as a type of myeloproliferative neoplasm, a group of cancers in which there is growth of abnormal cells in the bone marrow. ...
.
It is a
macrocyclic
Macrocycles are often described as molecules and ions containing a ring of twelve or more atoms. Classical examples include the crown ethers, calixarenes, porphyrins, and cyclodextrins. Macrocycles describe a large, mature area of chemistry.
...
protein kinase inhibitor. It mainly inhibits
Janus kinase 2
Janus kinase 2 (commonly called JAK2) is a non-receptor tyrosine kinase. It is a member of the Janus kinase family and has been implicated in signaling by members of the type II cytokine receptor family (e.g. interferon receptors), the GM-CSF rece ...
(JAK2) and
Fms-like tyrosine kinase 3\CD135 (FLT3).
Common side effects include diarrhea, low platelet counts, nausea, anemia, and swelling in legs.
Medical uses
Pacritinib in
indicated
In medicine, an indication is a valid reason to use a certain test, medication, procedure, or surgery. There can be multiple indications to use a procedure or medication. An indication can commonly be confused with the term diagnosis. A diagnosis ...
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.
[ ]
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 200mg 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.
The U.S.
Food and Drug Administration
The United States Food and Drug Administration (FDA or US FDA) is a List of United States federal agencies, federal agency of the United States Department of Health and Human Services, Department of Health and Human Services. The FDA is respon ...
(FDA) granted the application for pacritinib
priority review
Priority review is a program of the United States Food and Drug Administration (FDA) to expedite the review process for drugs that are expected to have a particularly great impact on the treatment of a disease. The priority review voucher program ...
,
fast track
The fast track is an informal English term meaning "the quickest and most direct route to achievement of a goal, as in competing for professional advancement". By definition, it implies that a less direct, slower route also exists.
Fast track or F ...
, and
orphan drug designations.
Society and culture
Names
Pacritinib is the
International nonproprietary name (INN).
References
External links
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Aminopyrimidines
Ethers
Antineoplastic drugs
Non-receptor tyrosine kinase inhibitors
Orphan drugs
1-Pyrrolidinyl compounds
Oxygen heterocycles
Nitrogen heterocycles
Ethanolamines
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