Merck ($MRK) walked away from yesterday's FDA panel review of the new hepatitis C drug boceprevir with a unanimous recommendation for marketing approval and a laundry list of suggested post-marketing studies to add to its to-do list. Next up is today's review of Vertex's telaprevir, which had an even better cure rate than boceprevir in clinical trials and which a top agency staffer suggested is already a favorite for approval as regulators close in on a pair of new therapies that can offer a whole new standard of care.
Backed by solid efficacy data and a ready response to concerns about anemia, the pharma giant's regulatory and development team's biggest challenge came as the experts questioned the absence of null responders--patients who had tried and failed treatment--in the study. That's something the panel wants the agency to include on the label, until Merck has a chance to gather data to see how they react to boceprevir. The panel also wants Merck to conduct studies on patients with HIV, minority groups and those who have had organ transplants, part of what panel chairperson Victoria Cargill referred to as the "long list" of studies to come.
Merck won out on the main issue, though, offering a knockout set of data demonstrating that boceprevir increased the cure rate from 38 percent for standard treatments to 65 percent. "It changes the game completely and hopefully continues to advance the field," says Cargill.
That was the view taken by Debra Birkrant, the director of FDA's antiviral-drugs division, according to a report in the Wall Street Journal. Looked at side by side, she said, boceprevir and telaprevir improve cure rates "to more than 30% above what we have today."
For Merck, the vote signals an open road to a new blockbuster franchise, which it badly needs. Sanford C. Bernstein's Geoffrey Porges notes that both drugs could generate up to $7 billion a year in just three years. That's critical for Vertex, which has been building up a sales organization as it prepares to roll out telaprevir in the U.S.