FDA Grants Orphan Drug Status to RedHill's Opaganib for Neuroblastoma

RedHill Biopharma Ltd., a specialized biopharmaceutical company, has announced that the U.S. Food and Drug Administration (FDA) has granted orphan-drug designation to its drug candidate, opaganib, for the treatment of neuroblastoma. This rare yet most common cancer in infancy accounts for about 15% of all pediatric cancer-related deaths. The orphan-drug designation can provide seven years of marketing exclusivity for opaganib if it gets FDA approval, alongside benefits such as accelerated development and review times, potential grants, and tax credits.

Neuroblastoma typically affects children under the age of five, with an annual incidence of about 650 new cases in the United States, accounting for approximately 10% of all childhood cancer cases. The standard treatment regimen includes surgery, chemotherapy, radiation therapy, and sometimes targeted or immunotherapy. However, there is an urgent need for new treatment options, as neuroblastoma continues to be a significant cause of pediatric cancer fatalities.

Opaganib is positioned as a promising oncological agent, having already received an orphan-drug designation for cholangiocarcinoma, a type of bile duct cancer. Dr. Mark Levitt, Chief Scientific Officer at RedHill, emphasized the broad oncology potential of opaganib, citing promising preliminary clinical data in solid tumors like prostate cancer and cholangiocarcinoma. The drug is also being studied for its potential to enhance hormone receptor pathway inhibition therapy, a prospect that will be explored in an upcoming Phase 2 study.

The neuroblastoma market is projected to reach nearly $1.5 billion by the mid-2030s, reflecting the critical need for effective treatments. Opaganib, a novel and orally administered small molecule, is in development for several indications, including viral, inflammatory, and diabetes-related conditions, alongside its oncology applications.

Neuroblastoma develops from neuroblasts, immature nerve cells found in the adrenal glands and nerve tissue along the spine, chest, abdomen, or pelvis. While the exact cause remains unclear, genetic mutations and abnormalities are known to play a role. In some cases, a genetic syndrome or family history may contribute, although most cases occur sporadically.

The prognosis for neuroblastoma varies widely, influenced by factors like the disease stage at diagnosis, the child’s age, and specific tumor characteristics. While the disease can regress spontaneously in very young children, it can also be aggressive and difficult to treat, leading to significant pediatric mortality.

Opaganib (ABC294640) is a first-in-class drug that inhibits sphingosine kinase-2 (SPHK2) and has demonstrated anticancer, anti-inflammatory, and antiviral activities. It targets multiple diseases including obesity-related conditions, gastrointestinal acute radiation syndrome (GI-ARS), COVID-19, Ebola, and other viruses. The drug works by inhibiting multiple pathways and inducing autophagy and apoptosis, thus disrupting viral replication.

The U.S. government has selected opaganib for evaluation in countermeasures against Acute Radiation Syndrome (ARS) and Sulfur Mustard exposure, funded by the National Institutes of Health (NIH) and the Biomedical Advanced Research and Development Authority (BARDA). Opaganib has shown antiviral activity against SARS-CoV-2 and several other viruses. It has demonstrated increased survival times in an Ebola virus study, marking it as the first host-directed molecule to show efficacy in this disease.

Opaganib has undergone various clinical trials, demonstrating safety and tolerability in over 470 participants. It has shown potential for pandemic preparedness and is in late-stage development for a range of conditions. With its promising preliminary data in oncology, RedHill Biopharma continues to advance opaganib through clinical development stages, aiming to address significant unmet medical needs in both pediatric and adult oncology.