FDA Approves New Drug to Treat Advanced Melanoma

The U.S. Food and Drug Administration (FDA) today approved a new drug for patients with late-stage melanoma, the most dangerous type of skin cancer.

Yervoy (ipilimumab) is a monoclonal antibody that blocks a molecule known as CTLA-4, which is thought to play a role in slowing down or turning off the body’s immune system, affecting its ability to fight off cancerous cells.

Yervoy, developed by Bristol-Myers Squibb Co., will initially be sold only for use in patients who have not responded to other treatments. The drug is administered intravenously.

Melanoma is the leading cause of death from skin disease. An estimated 68,130 new cases of melanoma were diagnosed in the United States during 2010 and about 8,700 people died from the disease, according to the National Cancer Institute.

“Late-stage melanoma is devastating, with very few treatment options for patients, none of which previously prolonged a patient’s life,” said Richard Pazdur, M.D., director of the Office of Oncology Drug Products in the FDA’s Center for Drug Evaluation and Research. "Yervoy is the first therapy approved by the FDA to clearly demonstrate that patients with metastatic melanoma live longer by taking this treatment."

Patients lived longer

Yervoy’s safety and effectiveness were established in a single international study of 676 patients with melanoma. All patients in the study had stopped responding to other FDA-approved or commonly used treatments for melanoma. In addition, participants had disease that had spread or that could not be surgically removed. 

The study was designed to measure overall survival, the length of time from when the treatment started until a patient's death. The randomly assigned patients received Yervoy plus an experimental tumor vaccine called gp100, Yervoy alone, or the vaccine alone.

 Those who received the combination of Yervoy plus the vaccine or Yervoy alone lived an average of about 10 months, while those who received only the experimental vaccine lived an average of 6.5 months.

While those benefits may appear modest, scientists say some people who took the drug several years ago when it was still in the early testing stages are still alive today.

"I think the vast majority of patients with metastatic melanoma should at some point try this agent," said Patrick Hwu, chairman of the department of melanoma medical oncology at the University of Texas MD Anderson Cancer Center in Houston.

Side effects

The drug can have severe side effects, including fatigue, diarrhea, skin rash, endocrine deficiencies (gland or hormone), and inflammation of the intestines (colitis). Severe to fatal autoimmune reactions were seen in 12.9 percent of patients treated with Yervoy. 

When severe side effects occurred, Yervoy was stopped and corticosteroid treatment was started. Not all patients responded to this treatment. Patients who did respond in some cases did not see any improvement for several weeks.

Due to the unusual and severe side effects associated with Yervoy, the therapy is being approved with a Risk Evaluation and Mitigation Strategy to inform health care professionals about these serious risks. A medication guide will also be provided to patients to inform them about the therapy's potential side effects, the FDA said.