EMA Grants Orphan Drug Status To ImmunoCellular's ICT-107

The European Medicines Agency has granted orphan drug designation for ImmunoCellular Therapeutics’ ICT-107 for the treatment of glioblastoma. The company has previously received orphan drug designation for ICT-107 in the U.S.

ImmunoCellular's Chief Executive Officer Andrew Gengos said, “Obtaining orphan designation in the EU for ICT-107 is an important achievement for our Company, as we believe that it adds value to our lead asset and creates additional development, commercialization and partnering opportunities in the European market.”

Orphan drug designation in the EU provides sponsor companies with a range of incentives which include a 10-year period of exclusivity, scientific advice during the course of product development, assistance in trial design, and access to a centralized review process. In addition, sponsor companies can also avail of fee reductions and tax incentives.

ICT-107 is an intradermally administered autologous vaccine comprised of the patient’s dendritic cells pulsed with six types of synthetic tumor-associated antigens: AIM-2, MAGE-1, TRP-2, gp100, HER-2, IL-13Rα2. The EMA designation was supported by the completion of a randomized, double-blind, placebo-controlled Phase II trial evaluating the drug’s safety and efficacy in patients with newly diagnosed patients with glioblastoma multiforme following resection and chemoradiation.

Glioblastomas (GBM) are typically highly malignant tumors that arise from the brain’s supportive tissue astrocytes. GBMs are usually found in the brain’s cerebral hemisphere but can occur anywhere in the brain or spinal cord. The tumor represents about 17% of all primary brain tumors and is found to occur more frequently in men than in women.

The company said it anticipates additional milestones in its ICT-107 program in the coming months. “We are formulating our EU regulatory strategy in preparation for our first meeting with the EMA.  We are completing our fourth-generation manufacturing process development, and plan to select a phase III manufacturer in the second half of this year,” Gengos said in the press release. He also said the company is expecting to hold an end-of-phase-II meeting with the FDA to discuss potential Phase III trial planning to start later in 2014.

 “We continue to believe that ICT-107 has significant therapeutic and commercial potential, and look forward to advancing our development and regulatory strategies,” Gengos said.