August 17th, 2011
02:34 PM ET
Many patients with the deadliest form of skin cancer got a new treatment option Wednesday, as the Food and Drug Administration gave its blessing to vemurafenib, sold by Genentech under the brand name Zelboraf. It was approved to treat patients whose tumors have a specific gene mutation known as BRAF (pronounced “bee-RAF), and with advanced disease or whose tumors cannot be removed through surgery.
“It’s good day for melanoma,” says Tim Turnham, president of the Melanoma Research Foundation. “We’ve gotten two new drugs this year, after 13 years of nothing.” The other drug, Yervoy, was approved in March.
About 70,000 patients a year are diagnosed with melanoma, Most cases are caught early, but if disease spreads beyond the original tumor site, the average lifespan is measured in months without treatment.
Zelboraf was approved under the FDA’s “priority review” process, and approved more than two months before the deadline under that review. Asked if there was a sense of urgency to the move, FDA spokeswoman Erica Jefferson replied by email, “FDA always works to get promising treatments to patients who have few or no treatment options as quickly as possible.”
Zelboraf is part of a trend known as “personalized medicine,” in which therapies are tailored to specific aspects of a patient’s disease – in this case, the BRAF mutation, which is found in approximately half of all melanoma tumors. The FDA approved Zelboraf in combination with a test that looks for the BRAF mutation. If it’s found, the patient could be prescribed medication, which comes in pill form – 4 pills taken twice daily.
Turnham says the effects can be spectacular. “Sometimes, before they start taking it, the [MRI] scan looks like someone’s been at ‘em with a paintball gun – all these dark spots. Literally two weeks later, you look at the scans and the spots are all gone.” Overall, results are somewhat less dramatic. In one of the trials reviewed by the FDA, 52% of patients saw their tumors shrink. In the other, 77% of patients were still living, compared to 64% of those taking only an older type of chemotherapy.
Zelboraf is currently approved for the treatment of advanced disease, but Turnham says some doctors will be tempted to use it for patients at earlier stages of disease. “At Stage 3, there’s a 50% chance of recurrence. If you give them this drug, could you keep it from coming back?” The decision won’t be easy. “You also have side effects, and it’s very expensive.”
While doctors are allowed to prescribe drugs for more than the specifically indicated conditions, cost could be a hurdle to wider use. A 6-month course of Zelboraf costs $56,000. According to Turnham, patients are likely to take the drug indefinitely, as long as their tumors are kept at bay.
Cost is not a new issue for new cancer drugs. Yervoy, for example, carries a reported price tag of $120,000 for a year of treatment.
According to the FDA, the most common side effects of Zelboraf include joint pain, rash, hair loss, fatigue, nausea, and skin sensitivity when exposed to the sun. Patients should avoid sun exposure when taking the drug.
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