Orphan Drug Designation for Opaxio for Malignant Brain Cancer

Cell Therapeutics announced that Opaxio (paclitaxel poliglumex) has been granted orphan drug designation by the FDA for the treatment of glioblastoma multiforme (GBM), a malignant brain cancer.

Orphan designation was granted based on preliminary activity seen from Phase 2 results of Opaxio when added to standard therapy (temozolamide [Temodar; Merck] plus radiation). In this study, progression-free and overall survival was encouraging among patients with GBM, including patients whose tumors expressed unmethylated MGMT. Current standard therapy is less effective in patients with tumors that have unmethylated MGMT, an important DNA repair enzyme. A randomized trial is now underway for patients with GBM with unmethylated MGMT comparing standard temozolamide and radiation to Opaxio and radiation.

Opaxio (paclitaxel poliglumex, CT-2103) is a biologically enhanced chemotherapeutic that links paclitaxel to a biodegradable polyglutamate polymer. When bound to the polymer, paclitaxel is inactive, potentially sparing normal tissue's exposure to high levels of paclitaxel and its associated toxicities. Blood vessels in tumor tissue, unlike blood vessels in normal tissue, are porous to macromolecules such as Opaxio. Based on preclinical studies, it appears that Opaxio is preferentially distributed to tumors due to their leaky blood vessels and trapped in the tumor bed. Once inside the tumor cell, enzymes metabolize the protein polymer, releasing active paclitaxel.

For more information call (206) 282-7100 or visit www.CellTherapeutics.com.