The U.S. Food and Drug Administration (FDA) has granted Orphan Drug designation to NanoSmart Pharmaceuticals’ novel formulation of dactinomycin for the treatment of Ewing sarcoma, a rare type of childhood bone cancer. The designation was granted on the basis of a plausible hypothesis that the novel formulation, which uses NanoSmart’s proprietary drug delivery platform, may be clinically superior to the original drug.
The new 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 sarcoma and other cancers but are limited by toxicity and often cause lifelong complications. The novel 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. ■