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Efaproxiral Granted Orphan Drug Status
The FDA may award orphan drug designation to drugs that target conditions affecting 200,000 or fewer U.S. patients per year and provide a significant therapeutic advantage over existing treatments. The orphan drug designation provides for U.S. marketing exclusivity for seven years following marketing approval by the FDA. Additionally, the designation qualifies a product for possible funding to support clinical trials, study design assistance from the FDA during development and other financial incentives.
"We are pleased to have been awarded the FDA's orphan drug designation for EFAPROXYN(TM)," said Michael E. Hart, Chief Executive Officer and President of Allos. "This designation is a significant milestone in the development of EFAPROXYN(TM), and, if approved for marketing in this indication, will become an important component of our commercialization strategy."
In February, Allos initiated a Phase 3, randomized, open-label, multi-center trial called ENRICH (Enhancing Whole Brain Radiation Therapy (WBRT) In Patients with Breast Cancer and Hypoxic Brain Metastases) designed to compare the effect of WBRT with supplemental oxygen with or without EFAPROXYN(TM) in women with brain metastases from breast cancer. Results from earlier trials showed a positive survival benefit and demonstrated a desirable safety profile in this patient population. EFAPROXYN(TM) is also currently being evaluated as a radiation sensitizer in patients with cervical cancer, (Stage IIIA/Stage IIIB) non-small cell lung cancer (NSCLC) and recurrent glioblastoma multiforme and as a possible chemo-enhancer enhancer for BCNU (carmustine) chemotherapy for the treatment of recurrent malignant glioma.
EFAPROXYN(TM) (efaproxiral) is the first synthetic small molecule designed to "sensitize" hypoxic (oxygen-deprived) areas of tumors prior to radiation therapy by facilitating the release of oxygen from hemoglobin, the oxygen-carrying protein contained within red blood cells, and increasing the level of oxygen in tumors. The presence of oxygen in tumors is an essential element for the effectiveness of radiation therapy in the treatment of cancer. By increasing tumor oxygenation at the time of treatment, EFAPROXYN(TM) has the potential to enhance the efficacy of standard radiation therapy. Unlike chemotherapeutics or other radiation sensitizers, EFAPROXYN(TM) does not have to cross the blood brain barrier or enter the tumor to be effective.
Source: Allos Therapeutics