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 a second drug product that uses NanoSmart's proprietary drug delivery platform.
The drug product is a formulation of dactinomycin for the treatment of Ewing's sarcoma, a rare type of childhood bone cancer. The FDA granted NanoSmart's Orphan Drug Designation on the basis of a plausible hypothesis that the novel formulation may be clinically superior to the original drug.
NanoSmart's second pipeline drug formulation consists of dactinomycin, a well-understood cancer drug, incorporated into a proprietary ANA-conjugated nanoemulsion. Cytotoxic agents like dactinomycin are known to be effective for Ewing's sarcoma and other cancers, but are limited by toxicity and often cause life-long complications. NanoSmart's formulation is expected to localize drug delivery to the tumor site, thereby increasing effectiveness at the tumor while simultaneously improving safety by reducing collateral damage to healthy tissues.
"The FDA's commitment to supporting drug development for rare and pediatric diseases has created opportunities for our industry to efficiently develop new therapies for patients that are most in need," stated James Smith, Ph.D., President of NanoSmart. "We are very pleased that the FDA has issued this designation and we look forward to commercializing these exciting drug delivery platforms."
The FDA Office of Orphan Products Development (OOPD) grants orphan status to promising drug therapies for rare diseases that affect less than 200,000 persons in the United States. This designation qualifies sponsor companies for certain development incentives, such as FDA fee waivers, substantial tax credits, access to grant funding for clinical studies, and potential for seven years market exclusivity upon successful completion of clinical trials and subsequent market approval.