BMS headquarters in NYC. Credit: BMS

The March 25 US Food and Drug Administration (FDA) approval of Bristol-Myers Squibb's Yervoy (ipilimumab) for metastatic melanoma was expected, but the breadth of the approval was not. Yervoy, a human monoclonal antibody targeting cytotoxic T-lymphocyte activator-4 (CTLA4) developed by the New York–based company, is the first agent to prolong survival in a phase 3 trial in metastatic melanoma (Nat. Biotechnol. 28, 763–764, 2010). The FDA has given the green light for Yervoy to be used in a first-line setting even though the pivotal trial included only individuals who had progressed on other treatments. It was “exactly the right decision,” says oncologist Mario Sznol of Yale University in New Haven, Connecticut, as no current first-line treatment improves survival in metastatic melanoma. FDA approval also allows patients who respond initially to Yervoy, but who later relapse, to receive another course of the drug. Sznol expects rapid adoption of the drug by oncologists, despite a $120,000 wholesale price tag for a single four-infusion course of treatment. “The first thing that has to be on your mind when somebody comes in with metastatic melanoma would be ipilimumab, based on the data,” Sznol says. Chris Schott, a pharma analyst at JP Morgan in New York, raised his earlier Yervoy estimates based on the higher-than-expected pricing, and now forecasts sales of $170 million in 2011, growing to $1.25 billion by 2015. Defending the price, Bristol-Myers Squibb spokesperson Sarah Koenig stresses the company's aggressive patient-assistance program. In the US, this “will enable coverage of virtually all, approximately 98%, of uninsured patients,” she writes in an e-mail. Another metastatic melanoma drug likely to win approval in the near term is PLX4032 (vemurafenib). PLX4032, a small-molecule inhibitor of mutant BRAF, was developed by the Berkeley, California–based Plexxikon, which was acquired by the Tokyo-based Daiichi Sankyo on April 4. PLX4032 produces higher response rates than Yervoy and an undisclosed survival benefit, although virtually all individuals taking the treatment relapse. So the drug probably won't hurt Yervoy sales even in the roughly half of metastatic melanoma patients who qualify for PLX4032, says Sznol, as most will end up taking Yervoy eventually. Plexxikon plans to apply for FDA registration this year.