FDA and EMA Approvals Facilitate Global Strategy for Development of Cytrx Orphan Drugs
March 31, 2014
CytRx Corporation, 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.
About Soft Tissue Sarcoma
Soft tissue sarcoma is a cancer occurring in muscle, fat, blood vessels, tendons, fibrous tissues and connective tissue, and can arise anywhere in the body at any age. According to the American Cancer Society, there are approximately 50 types of soft tissue sarcomas. In 2013 more than 11,400 new cases were diagnosed in the U.S. and approximately 4,400 Americans died from this disease. In addition, approximately 40,000 new cases and 13,000 deaths in the U.S. and Europe are part of a growing underserved market.
The widely used chemotherapeutic agent doxorubicin is delivered systemically and can be highly toxic, which limits its total exposure. Doxorubicin also has the potential to damage heart muscle at cumulative doses greater than 450 mg/m2. Aldoxorubicin attaches doxorubicin to a novel linker that binds directly and specifically to circulating albumin, the most plentiful protein in the bloodstream. Protein-hungry tumors concentrate albumin, thus increasing the delivery of the linker molecule with the attached doxorubicin to tumor sites. In the acidic environment of the tumor, but not the neutral environment of healthy tissues, doxorubicin is released from the linker. This allows for greater doses (3 ½ to 4 times) of doxorubicin to be administered without dose-limiting toxic side effects. In studies in cancer patients thus far there has been no evidence of clinically significant effects of aldoxorubicin on heart muscle, even at cumulative doses of drug well in excess of 2 g/m2.
See the press release here.