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FDA grants aldoxorubicin orphan drug status
CytRx Corp. announced that the FDA granted multiple orphan drug designation to aldoxorubicin for the treatment of patients with glioblastoma multiforme, small cell lung cancer and ovarian cancer.
Aldoxorubicin is a modified version of the chemotherapeutic agent doxorubicin, combined with a novel single-molecule linker that binds directly and specifically to circulating albumin. This combination allows patients to receive higher doses of doxorubicin while reducing its toxic side effects.
“The FDA’s decision to grant Orphan Drug designation for aldoxorubicin in these three new indications is a key milestone for the aldoxorubicin clinical development program and a clear reflection of the high unmet medical need for new treatments in these cancer types,” Steven A. Kriegsman, president and CEO of CytRx, said in a press release. “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 2 [glioblastoma multiforme] trial and our phase 2 Kaposi’s sarcoma trial in the first half of 2015.”
Aldoxorubicin is being evaluated in a global phase 3 clinical trial, examining its safety and efficacy as a second-line treatment for patients with soft tissue sarcoma under a special protocol assessment with the FDA.
In addition, CytRx has initiated two phase 2 clinical trials, assessing aldoxorubicin treatment for patients with late-stage glioblastoma multiforme and HIV-related Kaposi’s sarcoma. CytRx also plans to launch a global phase 2b trial in patients with relapsed small cell lung cancer, as well as a phase 1b combination study of aldoxorubicin plus gemcitabine as a potential precursor to a trial in patients with relapsed ovarian cancer.
The FDA Office of Orphan Products Development grants orphan drug designation to novel drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States.