First Gene-Targeted Drug for Melanoma Approved by FDA

Decision Opens New Doors to Long-Awaited Combination Trials

WASHINGTON, D.C.—Melanoma patients who have long faced a bleak treatment landscape are gaining a new approach for treating the deadliest form of skin cancer. The Food and Drug Administration today announced the approval of Zelboraf, known as vemurafenib or PLX4032 during testing, well ahead of the approval deadline. Zelboraf is the first drug to treat advanced melanoma by targeting a specific gene mutation.

Health care professionals have used targeted therapies to block the growth and spread of other cancers, including breast cancer, pancreatic cancer and non-small cell lung cancer. Zelboraf is the first approved drug to use this approach to treat melanoma.

“The FDA’s quick action on this drug approval is important because it gives melanoma patients a new way to fight this deadly disease,” said Timothy J. Turnham, the executive director of the Melanoma Research Foundation (MRF). “We have seen evidence that targeting gene mutations works. But it also demonstrates the very real challenges researchers still face in offering patients lasting results.”

Zelboraf is designed to kill cancer cells by blocking the mutated BRAF gene found in 40 to 60 percent of all melanoma patients. One trial found 84 percent of the advanced melanoma patients taking Zelboraf survived for at least six months, compared to 64 percent of the patients taking a traditional chemotherapy drug called dacarbazine. Zelboraf reduced the risk of death by 63 percent. (Source: American Society of Clinical Oncology. “Vemurafenib Improves Outcomes in BRAF-mutated Melanoma.” https://chicago2011.asco.org/ASCODailyNews/BRIM3.aspx)

While many patients experience a striking reprieve while taking this drug, for most, the melanoma becomes resistant, making recurrence of the cancer likely. On average, patients in Zelboraf’s clinical trials showed recurrence of tumor growth after seven months.

“The experience with Zelboraf shows that we’re on the right track but that we still have to discover a way to ensure the drug remains effective over time,” said Turnham. “The average advanced melanoma patient’s life expectancy is still being measured in months and by testing promising treatments in combinations, we think we can extend the lives of thousands of melanoma patients.”

Zelboraf is the second melanoma drug to be approved by the FDA this year and the first therapy approved that targets a specific gene mutation. The FDA’s announcement follows the March approval of Bristol-Myers Squibb’s Yervoy, a therapy designed to stimulate a person’s immune system to attack the cancer. Prior to 2011, the FDA had not approved a new melanoma treatment in 13 years.

Bristol-Myers Squibb and Zelboraf maker Roche agreed in June to partner to study the impact of using the treatments together. Similar combination approaches have proven effective in fighting diseases like HIV/AIDS, and most melanoma researchers agree studying combined treatments is likely to yield significant progress for patients. Having two advanced melanoma drugs on the market opens the door to explore more promising treatment combinations.

“We cannot allow bureaucratic or competitive hurdles to stand in the way of progress,” Turnham said. “We recognized the need for combination trials two years ago. During that time, nearly 20,000 melanoma patients have died waiting for a breakthrough.”

The MRF awards grants to support clinical research into melanoma’s causes and potential treatments. The organization supported early research into the BRAF gene mutation, work that laid the foundation for the development of Zelboraf.

For more information about the MRF and treatment options for melanoma, visit www.melanoma.org. If you are a patient who wants to explore the full range of treatment options, visit MRF’s new clinical trial finder at https://www.emergingmed.com/networks/MRF.

# # #

About Melanoma

Melanoma, the most serious type of skin cancer, is one of the fastest growing cancers in the United States and can strike men and women of all ages, all races and skin types. In fact, with a one in 50 lifetime risk of developing melanoma, nearly 69,000 Americans were expected to be diagnosed with the disease in 2010, resulting in 8,700 deaths or one person every hour. Melanoma is the most common form of cancer for young adults 25- to 29-years-old and the second most common cancer in adolescents and young adults 15- to 29-years-old.

About Melanoma Research Foundation

The Melanoma Research Foundation (MRF) is the largest independent, national organization devoted to melanoma in the United States. Committed to the support of medical research in finding effective treatments and eventually a cure for melanoma, the MRF also educates patients and physicians about prevention, diagnosis and the treatment of melanoma. The MRF is an active advocate for the melanoma community, helping to raise awareness of this disease and the need for a cure. The MRF’s website is the premier source for melanoma information seekers. More information is available at www.melanoma.org.