On February 23, 2015 Celldex Therapeutics reported that the U.S. Food and Drug Administration (FDA) has granted rindopepimut (Rintega) Breakthrough Therapy Designation for the treatment of adult patients with EGFRvIII-positive glioblastoma (GBM) (Press release Celldex Therapeutics, FEB 23, 2015, View Source [SID:1234501831]).

This application was based on data from the Phase 2 ReACT study in recurrent GBM, the Phase 2 ACT III study in newly diagnosed GBM and additional supportive Phase 2 studies. An international Phase 3 study of rindopepimut, called ACT IV, in newly diagnosed GBM completed enrollment (n=745) in December of 2014.

“The FDA’s decision to grant Breakthrough Designation underscores rindopepimut’s therapeutic potential for patients with glioblastoma,” said Anthony Marucci, Co-founder, President and Chief Executive Officer of Celldex Therapeutics. “These patients have extremely limited treatment options, with only three new drugs approved in more than twenty years. Emerging clinical data suggests that rindopepimut may offer an improvement over existing standard of care for EGFRvIII-positive patients. With continued positive data, we look forward to working closely with the FDA to support potential approval of rindopepimut as expeditiously as possible.”

According to the FDA, Breakthrough Therapy Designation is intended to expedite the development and review of drugs for serious or life-threatening conditions. The criteria for Breakthrough Therapy Designation require preliminary clinical evidence that demonstrates the drug may have substantial improvement on at least one clinically significant endpoint over available therapy.

Rindopepimut (Rintega) is an investigational immunotherapy that targets the tumor specific oncogene EGFRvIII. Patients with EGFRvIII-positive glioblastoma typically have a worse prognosis than the overall glioblastoma population, including poor long term survival.