According to a press release from Contract Pharma, RedHill Biopharma recently announced that its investigational therapy opaganib has earned the US Food and Drug Administration (FDA) Orphan Drug Designation as a treatment for neuroblastoma, a rare childhood cancer. This cancer, which affects immature cells of the nervous system, is believed to be responsible for 15% of deaths attributed to pediatric cancer.
This is the second Orphan Drug Designation that this therapy has earned, having previously received it for a different type of rare cancer, cholangiocarcinoma (bile duct cancer).
“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 U.S. government supported and Apogee conducted preclinical studies in various indications, including radioprotection, and also in combination with RedHill’s RHB-107. 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 2 study.” – Dr. Mark Levitt, Chief Scientific Officer, RedHill Biopharma
About Orphan Drug Designation
Orphan Drug Designation is a program from the FDA intended for therapeutics in development for the treatment of a disease that is regarded as rare, defined as affecting less than 200,000 people in the US. To qualify, the drug must either fulfill a currently unmet medical need or display potential advantages in efficacy or safety over currently available treatment options. This designation can confer several benefits to the recipient, such as more frequent communications with the FDA, the waiving of certain fees or taxes, and an exclusivity period lasting seven years if the drug is ultimately approved and introduced to the market.
About Neuroblastoma
Neuroblastoma is a type of cancer that appears in nerve tissue. Children are more frequently affected than adults. The direct cause of neuroblastoma is not known in most cases; in a small number of patients, an inherited mutation affecting the ALK gene has been implicated, but this only explains around two percent of cases at the most. Symptoms of neuroblastoma include a lump in the affected area which may be blue in color, bone and joint pain, loss of appetite, fatigue, and fever. Other symptoms vary depending on where the tumor is located; it typically originates in the adrenal glands but can also appear on the spine, neck, abdomen, or chest. Treatment approaches include immunotherapy, radiation therapy, chemotherapy, surgery, and stem cell transplant. Outcomes vary considerably; the five-year survival rate is 68% for patients between age one and fourteen. Learn more about neuroblastoma over at our partner site, Patient Worthy, click here.
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Last modified: September 20, 2024
