FDA Approves Zelboraf for Late-stage Melanoma

The US Food and Drug Administration (FDA) has approved its second drug designed to improve overall survival in melanoma patients this year; from Roche, Zelboraf (vemurafenib) treats patients with late-stage melanoma, the most deadly form of skin cancer.

“This has been an important year for patients with late-stage melanoma,” reported Richard Pazdur, MD, director of the Office of Oncology Drug Products in the FDA’s Center for Drug Evaluation and Research. “Zelboraf is the second new cancer drug approved that demonstrates an improvement in overall survival. In March, we approved Yervoy (ipilimumab), another new treatment for late-stage melanoma that also showed patients live longer after receiving the drug.”

The drug was engineered to treat only melanoma patients whose tumors produce a gene mutation known as BRAF V600E. Zelboraf was not tested in patients whose melanoma tests were negative for the mutation.

The BRAF protein is usually a part of the regulating cell growth, but in roughly 50% of patients with late-stage melanoma, it mutates. Zelboraf serves as a BRAF inhibitor, as it is able to block the function of the V600E-mutated BRAF protein.

Not only is Zelboraf’s approval vastly important to the melanoma community, but it is being approved with a novel test known as the cobas 4800 BRAF V600 Mutation Test, which will act as a companion diagnostic to aid in determining whether a melanoma patient’s cells have suffered from the BRAF V600E mutation.

The approval of the mutation test was based on the results from the same clinical study that got Zelboraf approved.

The creators of Zelboraf tested its safety and efficiency through an international trial of 675 patients suffering from late-stage melanoma with the BRAF V600E mutation who had not received previous therapy.

The participants of the study were placed into one of two groups and were administered either Zelboraf or dacarbazine, a separate anti-cancer treatment.

The median survival of patients who were administered Zelboraf has not been reached, as 77% of the patients are still alive; the median survival for patient who were given dacarbazine, however, was eight months, and only 64% are still alive.

“Today’s approval of Zelboraf and the cobas test is a great example of how companion diagnostics can be developed and used to ensure patients are exposed to highly effective, more personalized therapies in a safe manner,” stated Alberto Gutierrez, PhD, director of the Office of In Vitro Diagnostic Device Evaluation and Safety in the FDA’s Center for Devices and Radiological Health.

Zelboraf does not come without side effects. The most frequently reported side effects in patients taking the drug included joint pain, rash, hair loss, fatigue, nausea, and skin sensitivity when exposed to sunlight. Further, roughly 26% of patients in the study developed a skin-related cancer called cutaneous squamous cell carcinoma, which was treated with the aid of surgery.

The creators of Zelboraf warn that patients taking the drug should avoid sun exposure.