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Opaganib was given orphan drug designation for the treatment of neuroblastoma in children, which is rare, but the most common infancy malignancy.
Opaganib, a proprietary and investigational host-directed drug developed by Tel Aviv-based RedHill Biopharma, has been given orphan drug designation by FDA for treatment of neuroblastoma, a type of cancer that is the most common malignancy among infants, with a median age of 17 months at the time of diagnosis (1). RedHill announced the FDA decision in a press release on Aug. 26, 2024.
FDA has authority to grant a drug or biological product “orphan” status if such a product prevents, diagnoses, or treats a rare disease or condition (2). Neuroblastoma is rare among children overall, accounting for up to 10% of all childhood cancers in the United States, where it affects 11 to 13 children per million under the age of 15—though that number rises to 65 per million under one year old, the equivalent of approximately 650 new cases every year (1).
The cancer originates from neuroblasts (nerve cells) in the adrenal glands or the nerve tissue along the spine, chest, abdomen, or pelvis. It is usually found in children aged five or younger, but when diagnosed, it can be aggressive and difficult to treat, and is responsible for 15% of all pediatric cancer-related deaths, according to RedHill.
An orphan drug designation can qualify sponsors for incentives including tax credits for qualified clinical trials, exemption from user fees, and the potential for seven years of market exclusivity after approval, although it is a separate process from seeking approval or licensing, and FDA subjects drugs for rare diseases to the same rigors of the scientific review process as all other products (2).
This is the second orphan drug designation for opaganib in oncology, with the first having been for cholangiocarcinoma (CCA, or bile duct cancer), according to Mark Levitt, MD, PhD, RedHill’s chief scientific officer (1).
"Opaganib has broad oncology potential with promising preliminary clinical data in solid tumor cancers such as prostate cancer and CCA, and data from a range of US government supported and Apogee-conducted preclinical studies in various indications, including radioprotection, and also in combination with RedHill's RHB-107 (upamostat, another investigational drug),” said Levitt in the press release. “We also see such utility extending to the potential for opaganib to have a sensitizing effect in hormone receptor pathway inhibition therapy, which the company expects to test in a planned externally funded Phase II study."
Opaganib is an oral medication that RedHill said not only has anticancer properties, but also exhibits anti-inflammatory and antiviral activity, targeting everything from cancers to gastrointestinal- and obesity-related syndromes, sulfur mustard exposure, and COVID-19, Ebola, and other viruses with the potential to reach pandemic levels (1). It has also shown strong preclinical results in renal fibrosis.
1. RedHill Biopharma. RedHill's Opaganib Granted Orphan Drug Designation by the FDA for Childhood Cancer, Neuroblastoma. Press Release. Aug. 26, 2024.
2. FDA. Designating an Orphan Product: Drugs and Biological Products. FDA.gov/industry, updated Aug. 12, 2024 (accessed Aug. 26, 2024).
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