Perifosine (KRX-0401)

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Mechanism of action

From the NCI Drug Dictionary: An orally active alkyl-phosphocholine compound with potential antineoplastic activity. Targeting cellular membranes, perifosine modulates membrane permeability, membrane lipid composition, phospholipid metabolism, and mitogenic signal transduction, resulting in cell differentiation and inhibition of cell growth. This agent also inhibits the anti-apoptotic mitogen-activated protein kinase (MAPK) pathway and modulates the balance between the MAPK and pro-apoptotic stress-activated protein kinase (SAPK/JNK) pathways, thereby inducing apoptosis.

Preliminary data

Hodgkin lymphoma

  1. Guidetti A, Carlo-Stella C, Locatelli SL, Malorni W, Mortarini R, Viviani S, Russo D, Marchianò A, Sorasio R, Dodero A, Farina L, Giordano L, Di Nicola M, Anichini A, Corradini P, Gianni AM. Phase II Study of Perifosine and Sorafenib Dual-Targeted Therapy in Patients with Relapsed or Refractory Lymphoproliferative Diseases. Clin Cancer Res. 2014 Nov 15;20(22):5641-51. Epub 2014 Sep 19. link to original article PubMed

History of changes in FDA indication

  • 9/3/2009: Received FDA orphan drug designation for treatment of multiple myeloma.
  • 7/9/2010: Received FDA orphan drug designation for treatment of neuroblastoma.