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news.US-based NanoSmart Pharmaceuticals has received orphan drug designation from the US Food and Drug Administration (FDA) for a second drug product that uses the company's drug delivery platform.
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The new drug product is a formulation of dactinomycin indicated to treat Ewing’s sarcoma, a rare type of childhood bone cancer.
Orphan drug designation was awarded to the product based on a plausible hypothesis that the new formulation may be clinically superior to the original drug.
The drug formulation includes dactinomycin, a well-understood cancer drug, incorporated into a proprietary ANA-conjugated nanoemulsion.
Dactinomycin is a cytotoxic agent, which is known to be effective for Ewing’s sarcoma and other cancers, but is limited by toxicity and often causes life-long complications.
The 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.
NanoSmart president James Smith said: "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.
"We are very pleased that the FDA has issued this designation and we look forward to commercializing these exciting drug delivery platforms."