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FDA grants orphan drug designation to Anisina for neuroblastoma
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The FDA granted orphan drug designation to the chemotherapy drug Anisina for the treatment of pediatric patients with neuroblastoma, according to a press release from the drug’s manufacturer.
Anisina (Novogen Limited) — a small molecule belonging to the anti-tropomyosin family of compounds that is designed to inhibit the Tpm3.1 protein — received orphan drug status based on preclinical data. These data included preclinical studies conducted by The Kids’ Cancer Project in Sydney, Australia; The University of New South Wales in Sydney; The Nationwide Children’s Hospital in Columbus, Ohio; and Novogen.
The key findings from these studies showed that Anisina significantly improved the effectiveness of the standard-of-care microtubule targeting compound, vincristine, in an animal model of neuroblastoma.
“Obtaining the FDA-approved orphan drug designation for Anisina is an important strategy achievement for us,” Ian Phillips, interim chairperson of Novogen Limited, said in a press release. “It demonstrated the company’s commitment to bringing a drug to market which we hope will improve the outcome for children with neuroblastoma."
Novogen Limited plans to initiate a clinical trial based on the strength of the preclinical evidence.
“Given it is our intention to take this drug through to the clinic to treat childhood cancer, the designation affords additional guidance from the FDA in the design of our clinical trial program enhancing the efficiency and innovativeness of Anisina’s development,” Justine Stehn, PhD, BSc, anti-tropomyosin program director at Novogen Limited, said in a press release.
The FDA’s Office of Orphan Products Development grants orphan drug designation to novel drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States.