Date: 2016-03-21
Type of information: Granting of the orphan status in the EU
Product name: CA4P (fosbretabulin tromethamine)
Compound: fosbretabulin tromethamine
Therapeutic area: Cancer - Oncology - Rare diseases
Action mechanism: vascular disrupting agent/mitotic inhibitor/tubulin binder. CA4P (combretastatin-A4 phosphate or fosbretabulin) is an investigational vascular disrupting agent (VDA) product candidate, Upon administration, fosbretabulin is dephosphorylated to its active metabolite, combretastatin A4, which targets and binds to tubulin dimers and prevents microtubule polymerization, thereby resulting in mitotic arrest and apoptosis in endothelial cells. It also disrupts the engagement of the endothelial cell–specific junctional molecule vascular endothelial-cadherin (VE-cadherin) and so the activity of the VE-cadherin/?-catenin/Akt signaling pathway, which may result in the inhibition of endothelial cell migration and capillary tube formation. As a result of fosbretabulin's dual mechanism of action, the tumor vasculature collapses, resulting in reduced tumor blood flow and ischemic necrosis of tumor tissue.
Company: Oxigene, now Mateon Therapeutics (USA - CA)/Diamond BioPharm (UK)
Disease: neuroendocrine tumors
Latest news: * On March 25, 2016, Oxigene announced that the European Commission has granted orphan drug designation to CA4P (fosbretabulin tromethamine) for the treatment of gastro-entero-pancreatic neuroendocrine tumors (NETs). The company expects final data from its phase 2a clinical trial of CA4P in NETs to be available later in 2016. * On February 25, 2016, the Committee for Orphan Medicinal Products (COMP) has recommended the granting of an orphan designation for fosbretabulin tromethamine for treatment of gastro-entero-pancreatic neuroendocrine tumours. * On December 29, 2015, the FDA has granted orphan drug designation to CA4P for the treatment of neuroendocrine tumors.
Patents:
Submission of marketing authorization application USA :
Submission of marketing authorization application UE:
Withdrawal of marketing authorization application USA:
Withdrawal of marketing authorization application UE:
US authorization:
UE authorization:
Favourable opinion UE:
Favourable opinion USA:
Orphan status USA: 2015-12-29
Orphan status UE: 2016-03-21
Pediatric exclusivit _USA:
Pediatric exclusivity UE: OTC status: Other news:
