The U.S. Food and Drug Administration (FDA) has granted rare pediatric disease designation to opaganib1 for the treatment of neuroblastoma, a type of cancer most commonly affecting babies and young children

--

Rare pediatric disease designation provides for a Priority Review Voucher (PRV) subject to certain conditions

--

This new designation is in addition to opaganib's current neuroblastoma orphan drug designation, providing for potential benefits such as accelerated development and review times, FDA Prescription Drug User Fee Act (PDUFA) application fee waivers, tax credits and seven-years' marketing exclusivity, if approved

--

New preclinical data, presented at the 2026 American Association for Cancer Research (AACR) Annual Meeting, showed positive effects of opaganib as a potential add-on therapy in models of neuroblastoma and triple-negative breast cancer (TNBC)2

--

The neuroblastoma market is expected to be valued at approximately $3.5 billion in 20323

--

Opaganib is a novel, potentially broad acting, oral, small molecule drug with demonstrated safety & efficacy profiles4. It is in development for multiple oncology, viral (including Ebola virus disease (EVD)), inflammatory and diabetes and obesity-related indications