According to a story from empr.com, the Oncologic Drugs Advisory Committee, part of the US Food and Drug Administration (FDA), recently voted 16 to 0 against the investigational drug omburtamab as a treatment for neuroblastoma, a rare cancer that often affects children. More specifically, the vote reflects the committee’s conclusion that there was not sufficient evidence to demonstrate that the drug improved overall survival for patients with leptomeningeal/central nervous system metastases.
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 percent for patients between age one and fourteen. To learn more about neuroblastoma, click here.
Reasoning for Rejection
The Biologics License Application for omburtamab included data from two clinical studies. The panel decided to vote against the application because three issues regarding treatment efficacy:
- The panel concluded that the external control population did not provide a relevant comparison.
- Differences in survival rate could not be attributed to the drug following several sensitivity analyses.
- Lack of response rate data.
The BLA for omburtamab had originally been accepted in May of this year for priority review. The FDA officially rejected the treatment, based on the determination of the panel, at the end of November.
