ATC code None Synonyms R115777 PubChem CID 159324 | Legal status Investigational CAS Number 192185-72-1 IUPHAR/BPS 8025 | |
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Dr erba on tipifarnib for the treatment of older patients with aml
Tipifarnib (INN, proposed trade name Zarnestra) is a farnesyltransferase inhibitor that is being investigated in patients 65 years of age and older with newly diagnosed acute myeloid leukemia (AML). It inhibits the Ras kinase in a post-translational modification step before the kinase pathway becomes hyperactive. It inhibits prenylation of the CxxX tail motif, which allows Ras to bind to the membrane where it is active. Without this step the protein cannot function.
Contents
- Dr erba on tipifarnib for the treatment of older patients with aml
- What does tipifarnib mean
- Approval process
- Progeria
- References
It is also being tested in clinical trials in patients in certain stages of breast cancer. It is also investigated as a treatment for multiple myeloma.
For treatment of progressive plexiform neurofibromas associated with neurofibromatosis type I, it successfully passed phase I clinical trials but was suspended (NCT00029354) in phase II. The compound was discovered by and is under investigation by Johnson & Johnson Pharmaceutical Research & Development, L.L.C, with registration number R115777.
What does tipifarnib mean
Approval process
Tipifarnib was submitted to the FDA by Johnson & Johnson for the treatment of AML in patients aged 65 and over with a new drug application (NDA) to the FDA on January 24, 2005.
In June 2005, the FDA issued a "not approvable" letter for tipifarnib.
Progeria
It was shown on a mouse model of Hutchinson–Gilford progeria syndrome that dose-dependent administration of tipifarnib can significantly prevent both the onset of the cardiovascular phenotype as well as the late progression of existing cardiovascular disease.