FDA grants orphan drug designation to AVS100 for advanced melanoma
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The FDA granted orphan drug designation to AVS100 for the treatment of stage IIB through stage IV melanoma, according to the agent’s manufacturer.
AVS100 (Avstera Therapeutics) is a novel highly specific HDAC6 inhibitor with unique mechanisms in its ability to polarize macrophages to the anti-tumoral M1 phenotype, according to an Avstera Therapeutics-issued press release.
Macrophage differentiation and stabilization of M1 is important for maintaining anticancer responses. Studies conducted in preclinical animal models showed AVS100 reduced tumor growth and improved OS, according to the releasee.
“The FDA’s grant of this [orphan drug designation] for AVS100 highlights the significant unmet need ... for this patient population and the impact our therapy can have in potentially improving clinical outcomes,” Karthik Musunuri, CEO and co-founder of Avstera Therapeutics, said in the release. “Advanced-stage melanoma remains a complex and difficult cancer to treat with current approaches, and AVS100 has the potential to provide an opportunity to potentially reduce the disease burden shared by these patients.”
The FDA 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. The designation allows manufacturers to qualify for various incentives, including tax credits for qualified clinical trials and — upon regulatory approval — 7 years of market exclusivity.