NanoSmart's ANA-conjugated liposomal doxorubicin receives FDA Orphan Drug Designation

NanoSmart^® Pharmaceuticals, Inc., a private pharmaceutical company developing nanoparticle drug delivery platforms, has received Orphan Drug Designation from the Food and Drug Administration (FDA) for one of its lead candidates, antinuclear antibody (ANA) conjugated liposomal doxorubicin. The drug product is initially intended to treat Ewing's sarcoma, a rare type of cancer that develops in or around children's bones, with potential expansion into other indications.

NanoSmart is developing an improved, ANA-targeted, liposomal formulation of doxorubicin that focuses drug delivery at the tumor site. Non-clinical testing of NanoSmart's formulation has been very promising, revealing its potential to improve the safety and efficacy of liposomal doxorubicin. Because ANA conjugation enables the drug to bind to areas of necrosis that are present in solid tumors, NanoSmart anticipates expanding into additional pediatric indications.

"We are very pleased that the FDA has approved our request for orphan drug designation," explains Dr. James Smith, President of NanoSmart Pharmaceuticals, "This is an important regulatory milestone and the incentives enabled by this designation will allow us to continue with a very cost-effective commercialization strategy."

The FDA grants orphan status to drug therapies for rare diseases that affect less than 200,000 persons in the United States, and to sponsors that provide a plausible hypothesis that their drug formulation may be clinically superior to the same drug that is already approved for the same orphan indication. Sponsor companies qualify for certain development incentives, such as FDA fee waivers, substantial tax credits, access to grant funding for clinical studies, and potential for a period of market exclusivity upon approval.