The FDA has approved the novel drug ipilimumab for the treatment of patients with metastatic melanoma, marking another milestone for immunotherapy in the treatment of cancer.
The drug, which Bristol-Myers Squibb will market under the name Yervoy, “is the first therapy approved by the FDA to clearly demonstrate that patients with metastatic melanoma live longer by taking this treatment," Richard Pazdur, MD, director of the Office of Oncology Drug Products in the FDA's Center for Drug Evaluation and Research, said in a press release.
The drug is indicated for patients with late-stage melanoma, the most deadly form of skin cancer. The disease has been on the rise in the United States for at least 30 years, with 68,130 new cases and about 8700 deaths last year.
The approval of ipilimumab is the latest breakthrough for immunotherapy, which cancer researchers have chased for more than a century amid setbacks and skepticism. Last April, the prostate cancer vaccine Provenge was approved for the treatment of prostate cancer.
Ipilimumab, which is administered intravenously, is a monoclonal antibody that blocks the cytotoxic T-lymphocyte antigen (CTLA-4) molecule, which researchers suspect of slowing the body’s immune system.
The FDA said its decision to approve the drug was based on an international study of 676 patients for whom other approved therapies were no longer effective and who were not candidates for surgery. Patients were randomly assigned to receive ipilimumab plus the experimental tumor vaccine gp100, ipilimumab alone, or the vaccine alone.
Patients whose treatment included ipilimumab lived an average of 10 months, while those who received gp100 alone lived an average of 6.5 months, the FDA said.
Of those patients treated with ipilimumab, 12.9% exhibited severe-to-fatal autoimmune reactions, with fatigue, diarrhea, skin rashes, endocrine deficiencies, and colitis among the most common adverse events.
As a result, the FDA said it is requiring as part of its approval a risk management evaluation and mitigation strategy (REMS) to better inform healthcare providers of the risks of the drug and a medication guide for patients.