Ziopharm gets EU Orphan nod for palifosfamide

USA-based Ziopharm Oncology says that the European Medicines Agency's (EMEA) Committee for Orphan Medicinal Products (COMP) has issued a positive opinion regarding the firm's application for Orphan Medicinal Product designation for palifosfamide in the treatment of soft-tissue sarcoma.

"There is a significant unmet need for additional treatments to address soft-tissue sarcoma beyond locally-effective surgery," said Jonathan Lewis, chief executive. "We are pleased to so soon have orphan drug status in Europe to augment the Orphan Drug Designation received in the USA earlier this year as we initiate patient treatment in our Phase II, randomized controlled trial in STS," he added.

Palifosfamide, the active moiety of ifosfamide, is a bi-functional alkylator that causes irreparable inter-strand DNA cross-linking, resulting in cell death. The drug candidate is equal to, or more active, than ifosfamide in diverse cancer models, and is directly active against cancer cells but not metabolized to acrolein or chloroacetaldehyde, which cause bladder or central nervous system toxicities. The drug is completing a Phase II trial in advanced soft-tissue and bone sarcomas and a Phase I combination study with Adriamycin (doxorubicin). The company has recently initiated treatment in a randomized, controlled Phase II trial of doxorubicin and doxorubicin plus palifosfamide in the front- and second-line setting of soft-tissue sarcoma.