INV724 Earns FDA Orphan Drug and Rare Pediatric Drug Designations in Neuroblastoma

3D illustration of brain anatomy: © PIC4U - stock.adobe.com

• The FDA has granted rare pediatric disease designation (RPDD) and orphan drug designation (ODD) to INV724 for the treatment of neuroblastoma.
• INV724 is a novel GD2- and B7-H3–targeting bispecific antibody.
• With RPDD and ODD, Invenra, the sponsor, is eligible for several benefits for drug development and review.

INV724, a novel bispecific antibody, has been granted FDA RPDD and ODD for the treatment of neuroblastoma.¹

With these designations, Invenra, the sponsor of INV724, is eligible for several benefits to facilitate drug development and review. With ODD, the sponsor can be qualified for tax credits for clinical trials, exemption from user fees, and a potential 7 years of market exclusivity following drug approval.² The RPDD incentivizes drug development for rare pediatric diseases by offering a priority review voucher.

If INV724 is approved, the sponsor may be qualified for a voucher to receive priority review for a different product.³

"Anti-GD2 antibodies are a key component in treating neuroblastoma, but their use is often limited by severe pain due to nerve binding. Our studies indicate that INV724 binds effectively to neuroblastoma cells while sparing nerves, offering a significant advantage over existing therapies. Our next goal is to transition from laboratory research to clinical trials, aiming to provide effective treatment with fewer side effects," said Paul Sondel, MD, PhD, professor of pediatrics and human oncology at the University of Wisconsin, in a press release.¹

INV724 is a product of Invenra’s B-Body® Bispecific Platform and targets GD2 and B7-H3 tumor antigens with specific for neuroblastoma.1 This platform enables higher yield than other bispecific platforms. B-Body bispecifics are also suited for subcutaneous administration due to their high solubility, stability, and low viscosity.⁴

"Invenra is deeply committed to advancing INV724 as a potentially transformative treatment for children with neuroblastoma. The FDA's RPDD and ODD designations not only validate our innovative approach but also give us the critical support to fast-track its development in collaboration with potential partners. We are eager to bring this therapy to patients who need it most," said Roland Green, chief executive officer of Invenra, in the press release.¹

REFERENCES:

1. Invenra’s novel antibody INV724 receives rare pediatric disease and orphan drug designations from the US FDA for neuroblastoma treatment. News release. Invenra Inc. August 23, 2024. Accessed August 27, 2024. https://tinyurl.com/32uedrxp

2. Designating an orphan product: drugs and biological products. FDA. Updated August 12, 2024. Accessed August 27, 2024. https://tinyurl.com/24muw8am

3. Rare pediatric disease designation and priority review voucher programs. FDA. Updated May 28, 2024. Accessed August 27, 2024. https://tinyurl.com/yj6dxma7

4. Our Science. Invenra Inc. Accessed August 27, 2024. https://invenra.com/our-science