FDA grants orphan drug designation to RedHill's neuroblastoma treatment

Opaganib, a proprietary and experimental host cell-targeting drug developed by Tel Aviv-based RedHill Biopharma, has received orphan drug designation from the FDA for the treatment of neuroblastoma, a type of cancer that is the most common malignancy in infants, where the average age at diagnosis is 17 months (1). RedHill announced the FDA decision in a press release on August 26, 2024.

The FDA has the authority to grant orphan drug designation to a drug or biological product if such a product prevents, diagnoses, or treats a rare disease or condition (2). Neuroblastoma is rare in children overall, accounting for up to 10% of all cancers in the United States. It affects 11 to 13 out of a million children under the age of 15—but in children under one year of age, this number rises to 65 out of a million, which equates to about 650 new cases per year (1).

The cancer arises in neuroblasts (nerve cells) in the adrenal glands or in nerve tissue along the spine, chest, abdomen or pelvis. It usually occurs in children ages five or younger, but when diagnosed, it can be aggressive and difficult to treat, and is responsible for 15% of all cancer-related deaths in children, according to RedHill.

Orphan drug status entitles sponsors to incentives such as tax credits for qualified clinical trials, exemption from royalties, and possible seven years of market exclusivity after approval. However, this is a separate process from applying for approval or licensure. In addition, the FDA subjects rare disease drugs to the same rigorous scientific review processes as all other products (2).

According to Mark Levitt, MD, PhD, Chief Scientific Officer of RedHill, this is the second orphan drug designation for opaganib in oncology. The first approval was for cholangiocarcinoma (CCA or bile duct cancer) (1).

“Opaganib has broad oncology potential with promising preliminary clinical data in solid tumors such as prostate cancer and CCA, as well as data from a number of U.S. government-supported preclinical studies conducted by Apogee in various indications, including radiation protection, and also in combination with RedHill's RHB-107 (upamostat, another investigational drug),” Levitt said in the press release. “We also see such utility in that opaganib may have a sensitizing effect in hormone receptor pathway inhibition therapy, which the company plans to test in a planned, externally funded Phase II trial.”

Opaganib is an oral drug that RedHill says not only has anti-cancer properties, but also shows anti-inflammatory and antiviral activity, targeting everything from cancer to gastrointestinal and obesity-related syndromes, mustard gas exposure, as well as COVID-19, Ebola, and other viruses with the potential to reach pandemic proportions (1). It has also shown strong preclinical results in renal fibrosis.

References

1. RedHill Biopharma. FDA grants orphan drug designation to RedHill's opaganib for the treatment of childhood cancer and neuroblastoma. Press release. August 26, 2024.
2. FDA. Orphan Product Designation: Drugs and Biological Products. FDA.gov/Industryupdated on August 12, 2024 (accessed August 26, 2024).