FDA Grants Triple Orphan Designations To CytRx Lead Drug

Biopharmaceutical R&D firm CytRx announced that its lead drug candidate aldoxorubicin has been awarded multiple Orphan Drug Designations by the U.S. Food and Drug Administration (FDA) in three indications. These indications include glioblastoma multiforme (GBM), small cell lung cancer, and ovarian cancer.

Aldoxorubicin is the company’s version of the commonly used chemotherapeutic agent doxorubicin. Due to its high toxicity and systemic delivery, doxorubicin’s dosage is limited, in turn making it less effective in treatment. Aldoxorubicin comprises doxorubicin and a novel single-molecule linker that specifically binds to a common bloodstream protein, albumin. This makes the drug highly attractive to tumors that crave protein, allowing for the delivery of doxorubicin to acidic tumor sites while sparing healthy tissue. Therefore, Aldoxorubicin can be delivered in doses 3.5 to 4 times the normal dose of doxorubicin in cancer patients.

Steven A. Kriegsman, CytRx President and CEO, said, “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… We look forward to reporting top-line results from both our Phase 2 GBM trial and our Phase 2 Kaposi's sarcoma trial in the first half of 2015.”

The company is currently conducting a pivotal global Phase 3 trial investigating aldoxorubicin in soft tissue sarcoma (STS). Two separate Phase 2 clinical trials are also assessing the drug in patients with late-stage GBM and in HIV-related Kaposi's sarcoma. The company plans to initiate a Phase 1B combination study of the drug and gemcitabine in order to determine the combo’s potential before launching a clinical trial in relapsed ovarian cancer. CytRx recently announced the start of a global Phase 2B clinical trial for aldoxorubicin in extensive-stage small cell lung cancer (SCLC).

Should the drug receive approval in its orphan indications, CytRx stands to receive seven years of marketing exclusivity in the U.S. in addition to other incentives, such as tax credits and exemption from regulatory fees.