EpiCept Corporation has submitted a New Drug Application (NDA) to the US Food and Drug Administration (FDA) for Ceplene (histamine dihydrochloride). EpiCept is seeking approval for Ceplene, administered concomitantly with low-dose interleukin-2 (IL-2), for the remission maintenance and prevention of relapse in patients with Acute Myeloid Leukaemia (AML) in first complete remission. Ceplene was approved for this indication by the European Commission in October 2008 and is currently available in the United Kingdom, Germany and Austria. Ceplene is expected to be available throughout most of the European Union within twelve months.

In its application, EpiCept requested priority review of the NDA. If granted, priority review should result in an FDA decision date in late December 2010. A decision with respect to the granting of priority review must be made within 45 days of the filing date. Ceplene has been granted orphan drug status in the United States, providing seven years' marketing exclusivity from the NDA approval date.

"This filing is a very important milestone in our strategy for Ceplene, and we are delighted to submit the NDA consistent with the timetable we publicly announced," stated Jack Talley, president and chief executive officer of EpiCept. "There are an estimated 9,000 deaths caused by AML each year in the US with no effective maintenance therapy currently available. We believe that Ceplene, if approved, would favourably impact the prognosis of patients with this lethal disease."

The NDA submission is supported by a robust body of evidence demonstrating a significant clinical benefit of Ceplene/IL-2 for remission maintenance in AML as compared with the standard-of-care, which is no treatment. The pivotal phase III study was conducted in ten countries and included 320 randomized patients. The data demonstrated that patients with AML in complete remission who received up to 18 months of treatment with Ceplene plus low-dose IL-2 experienced a significantly longer period of leukaemia-free survival (LFS) compared to the standard-of-care. The difference between the treated and control group was statistically significant (p<0.008). Ceplene is the only medical therapy shown to extend LFS in this deadly form of leukaemia.

At the request of the FDA, EpiCept has included in its NDA the results of two meta-analyses to help isolate Ceplene's efficacy. The first analysis confirmed the lack of clinical efficacy of IL-2 monotherapy as remission maintenance for AML patients and the second demonstrated the contribution of Ceplene in this indication when given in conjunction with IL-2. These two meta-analyses were conducted by independent statisticians and support the conclusions of the pivotal phase III trial.

If priority review is not granted, the FDA has up to 60 days to accept the filing for review and assign an FDA decision date. In addition to the NDA filing in the US, a New Drug Submission for Ceplene is currently under active review by Health Canada, which is expected to complete its review and provide a decision by the fourth quarter of 2010. Ceplene is also under review by the Israeli Ministry of Health. As noted earlier, Ceplene is approved in thirty countries in Europe.

EpiCept is focused on the development and commercialization of pharmaceutical products for the treatment of cancer and pain.