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FDA grants orphan drug designation to dactinomycin formulation for Ewing's sarcoma
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The FDA granted orphan drug designation to a formulation of dactinomycin for the treatment of children with Ewing’s sarcoma, according to a press release from the drug’s manufacturer.
The drug product is a formulation of dactinomycin incorporated into a proprietary antinuclear antibody (ANA)-conjugated nanoemulsion (NanoSmart Pharmaceuticals). This is the second drug product that uses NanoSmart Pharmaceuticals’ proprietary drug delivery platform.
Dactinomycin is known to be effective for Ewing’s sarcoma and other cancers but can be limited by toxicity and long-term adverse events, according to the press release. This formulation intends to localize drug delivery to the tumor site to increase effectiveness and improve safety by reducing damage to local tissues.
The FDA granted NanoSmart’s formulation of dactinomycin orphan drug designation based on a plausible hypothesis that it would be clinically superior to the original drug.
In February, the FDA granted orphan drug designation to ANA-conjugated liposomal doxorubicin (NanoSmart Pharmaceuticals) for the treatment of patients with Ewing’s sarcoma.
"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 who are most in need," James Smith, PhD, president of NanoSmart Pharmaceuticals, said in a press release. "We are very pleased that the FDA has issued this designation and we look forward to commercializing these exciting drug delivery platforms."