FDA grants orphan drug designation for ICT-107

Jun 11 2010

ImmunoCellular Therapeutics (OTCBB: IMUC) announced today that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation for ICT-107, the company's dendritic cell-based cancer vaccine candidate which targets glioblastoma multiforme (GBM). As a result of the orphan drug status, ImmunoCellular will be eligible to receive a number of benefits, including access to grant funding for clinical trials, tax credits, accelerated FDA approval and allowance for marketing exclusivity after drug approval for a period of up to seven years. US orphan drug designation is granted to companies with products aimed at treatment of a rare disease or condition that affects fewer than 200,000 Americans.

“We are pleased that FDA has recognized the potential that ICT-107 has to treat GBM and has granted us orphan drug status”

A recent Phase I clinical study of ICT-107 in GBM revealed that newly diagnosed patients who received the vaccine demonstrated a median progression-free survival (PFS) of 17.7 months after surgery, compared with the historical median PFS of 6.9 months observed with standard treatment with surgery, radiation and chemotherapy. Seven of the 16 patients (44%) who participated in the study went on to live with no disease progression with an average time over 2 years, significantly better than historical data of less than 15% disease free survival. The drug has exceeded its competitors in the overall survival rate in newly diagnosed GBM patients by demonstrating an 80% survival, which is about three times larges than the historical control.

"We are pleased that FDA has recognized the potential that ICT-107 has to treat GBM and has granted us orphan drug status", said ImmunoCellular Therapeutics' president and CEO said Manish Singh, Ph.D. "The designation should facilitate a shorter time period to approval and reflects the FDA's views on the importance of developing a treatment which will improve survival outcomes for those afflicted with this terrible disease."

The 12-month disease-free survival from the time of surgery was 75% with ICT-107, compared with a historical control survival rate of 26.9%, and the 18-month disease-free survival with ICT-107 was 49.2%, compared with 18.4% historically. Safety data for ICT-107 also compared favorably to current treatments: no serious adverse events were reported and minor side effects were limited to fatigue, skin rash and pruritis.

SOURCE ImmunoCellular Therapeutics, Ltd.