CytRx’s aldoxorubicin receives European orphan medicinal product designation for treatment of advanced soft tissue sarcomas

Mar 31 2014

CytRx Corporation (Nasdaq: CYTR), a biopharmaceutical research and development company specializing in oncology, today announced that aldoxorubicin has received orphan medicinal product designation from the European Commission for the treatment of advanced soft tissue sarcomas. The designation is to encourage the development of drugs which may provide significant benefit to patients suffering from rare diseases.

"There is a significant unmet medical need for drugs to treat advanced soft tissue sarcomas," said Steven A. Kriegsman, CytRx President and CEO. "This European designation, together with our U.S. orphan designation, will facilitate our global development of aldoxorubicin to help improve the available treatment options for cancer patients suffering from this aggressive and difficult to treat cancer."

Aldoxorubicin is the Company's lead product candidate which combines the chemotherapeutic agent doxorubicin with a novel linker-molecule that binds specifically to albumin in the blood to allow for delivery of greater amounts of doxorubicin (3.5 to 4 times) without several of the major dose-limiting toxicities seen with administration of doxorubicin alone. Aldoxorubicin has been granted orphan drug designation by the Office of Orphan Product Development of the U.S. Food and Drug Administration (FDA) for the treatment of patients with soft tissue sarcomas and pancreatic cancer.

Under European Medicines Agency (EMA) guidelines, Orphan Medicinal Product Designation provides up to 10 years of market exclusivity if the product candidate is approved for marketing in the European Union and the orphan designation is maintained. Orphan status also permits EMA assistance in optimizing the candidate's clinical development through participation in clinical trial design and preparation of the product marketing application. Additionally, a drug candidate designated by the EMA as an Orphan Medicinal Product may qualify for a reduction in regulatory fees as well as a European Union-funded research grant.

Aldoxorubicin is currently being evaluated in a global pivotal Phase 3 trial under a special protocol assessment (SPA) as a therapy for patients with soft tissue sarcomas whose tumors have progressed following treatment with chemotherapy. CytRx also recently initiated a Phase 2 clinical trial with aldoxorubicin in patients with late-stage glioblastoma (brain cancer), and a Phase 2 clinical trial in HIV-related Kaposi's sarcoma.