Celldex Therapeutics Announces Orphan Drug Designation In The European Union For Rindopepimut, A Novel EGFRvIII Vaccine For Glioblastoma

Celldex Therapeutics, Inc. (Nasdaq: CLDX) today announced that the European Medicines Agency (EMA) has granted orphan drug designation for rindopepimut for the treatment of Glioblastoma (GB). GB is the most common and aggressive form of brain cancer. Rindopepimut is an immunotherapy that targets the tumor-specific oncogene (a growth promoter), EGFRvIII.

“Obtaining orphan designation for rindopepimut in the European Union (EU) is an important regulatory milestone for Celldex,” said Anthony S. Marucci, President and Chief Executive Officer of Celldex Therapeutics. “The benefits include 10 years of market exclusivity from product launch in the EU, fee reductions, as well as access to the central authorization procedure. We have already been granted Orphan Drug designation for this program by the FDA, which allows for 7 years of market exclusivity from product launch in the U.S. as well as Fast Track designation to accelerate the review of rindopepimut.”

“These orphan drug designations support our global development strategy for rindopepimut and our goal of providing improved therapies for patients with EGFRvIII positive glioblastoma,” said Dr. Thomas Davis, Senior Vice President and Chief Medical Officer of Celldex Therapeutics. “Our clinical experience to date with rindopepimut has demonstrated its potential in prolonging overall survival in front-line EGFRvIII expressing glioblastoma, where limited treatment options exist. This activity, paired with a favorable safety profile, underpins our plans to move forward with the initiation of a Phase 3 study of this program by year-end 2011.”

After consultations with both U.S. and EU regulatory authorities, Celldex has finalized the clinical protocol for the Phase 3 randomized, double-blind study of rindopepimut in GB. The primary endpoint of the study will be overall survival. The study, named ACT IV, is expected to enroll up to 374 patients with newly-diagnosed, fully resected, EGFRvIII expressing GB from over 150 clinical sites internationally. Enrollment is expected to begin in the fourth quarter of 2011.