CytRx, a biopharmaceutical R&D company specializing in oncology, has announced that the FDA has granted multiple Orphan Drug designations for the company's lead drug candidate, aldoxorubicin, in three indications: glioblastoma multiforme (GBM), small cell lung cancer and ovarian cancer. Aldoxorubicin is CytRx's modified version of the widely-used chemotherapeutic agent, doxorubicin.
Steven A. Kriegsman, CytRx president and CEO, said, "These designations are also a testament to the team's regulatory and development expertise and part of our core strategy to bring aldoxorubicin to patients worldwide as rapidly as possible. We look forward to reporting top-line results from both our phase II GBM trial and our phase II Kaposi's sarcoma trial in the first half of 2015."
In the U.S., under the Orphan Drug Act, the FDA's Office of Orphan Products Development (OOPD) grants orphan drug status to a drug intended to treat a rare disease or condition, which is generally a disease that affects fewer than 200,000 individuals in the country. Upon approval, if received, the designation provides aldoxorubicin with certain benefits, including seven years of U.S. market exclusivity in the specified indications if the sponsor complies with certain FDA requirements. Additional incentives for the sponsor include tax credits related to qualified clinical trial expenses and a possible exemption from FDA application fees.
Aldoxorubicin currently is being studied in a pivotal global phase III clinical trial evaluating the efficacy and safety of aldoxorubicin as a second-line treatment for patients with soft tissue sarcoma (STS) under a Special Protocol Assessment with the FDA. CytRx also is evaluating aldoxorubicin in two phase II clinical trials, one in patients with late-stage GBM and the other in HIV-related Kaposi's sarcoma. The company expects to start a global phase IIb trial in patients with relapsed small cell lung cancer this month and is undertaking a phase Ib combination study of aldoxorubicin plus gemcitabine as a potential precursor to a trial in relapsed ovarian cancer.