FDA Gives Orphan Drug Status To Keryx Perifosine

Keryx said that currently, Perifosine is in Phase 3 clinical development for refractory advanced colorectal cancer and multiple myeloma, both of these Phase 3 programs being conducted under special protocol assessment (SPA) agreements with the FDA with fast track designations obtained for both indications. Perifosine is also in Phase 1 and 2 clinical development for several other tumor types.

The Phase 1 data of Perifosine in recurrent pediatric solid tumors, including neuroblastoma, presented at the 46th Annual Meeting of the American Society of Clinical Oncology (ASCO), demonstrated Perifosine to be safe and well tolerated in children with advanced solid tumors and that Perifosine may have antitumor clinical activity as a single agent in neuroblastoma.

In a preclinical study, Perifosine showed reduction in neuroblastoma cell survival, slowed or regressed tumor growth, and increased survival in mice bearing neuroblastoma tumors.

A decreased level of activated Akt was also observed in Perifosine-treated neuroblastoma cells and xenograft tumors.

Ron Bentsur, CEO of Keryx, said: “The Orphan Drug designation is an important component of our development plan for Perifosine in neuroblastoma, an indication where no FDA approved therapies currently exist.

“We are currently exploring next steps for the development of Perifosine in this indication which we hope, ultimately, could provide a new treatment option for children and infants suffering with neuroblastoma.”