The FDA often finds a few flies in the ointment when it digs through new drug applications, but in the case of Cambridge, MA-based Vertex Pharmaceuticals (NASDAQ: [[ticker:VRTX]]), its new hepatitis C drug looks a bit better under close scrutiny. Shares of Vertex climbed more than 10 percent after FDA briefing documents on the new treatment were posted online this morning.
The U.S. drug regulator said today that its analysis of data submitted from Vertex said the cure rate climbed to as high as 79 percent among patients getting their first-round of treatment with Vertex’s telaprevir and two standard drugs—a smidge higher than the 75 percent cure rate previously reported by the company. That finding, from a study known as Advance, was released in a briefing document posted online by FDA staff ahead of a public advisory committee in Silver Spring, MD this Thursday. The FDA did, however, cite two main side effects—rash and anemia—in its safety review, and a question about the rash side effect is the first one that FDA is asking its expert advisory panel on Thursday.
The FDA’s detailed review, and the input from the advisory panel, are pivotal events in the history of Vertex, which has spent more than 20 years on its fabled quest to create what one author famously described as a “billion-dollar molecule.” If Vertex can persuade the FDA that its new medicine for hepatitis C deserves a spot on the market—and most analysts predict it will—then it will be in position to make billions of dollars and change the standard of care for this chronic liver infection that affects an estimated 3 million people in the U.S. Vertex has studied telaprevir in more than 40 studies involving 4,000 patients. It lost more than $700 million last year as part of its pre-launch push, which analysts expect will yield more than $2 billion in U.S. sales within a couple years.
Vertex shares rose $6.28, or about 13 percent, to $54.32 a share at 10:50 am Eastern time today after the FDA documents were made public. The FDA has until May 23 to make its decision on whether to clear the Vertex drug for sale in the U.S., so investors are clearly wagering based on the staff review that the new drug will soon get the green light.
The agency did hone in on side effects, but not in nearly as harsh a way as it often does with applications from other companies. Rash was reported in 56 percent of patients on the Vertex drug and a combination of two other standard meds, while only 32 percent had rash on the other two drugs alone, the FDA said. The rash was mostly mild to moderate, the FDA said, although it was severe 1 percent of the time, and caused 6 percent of patients in clinical trials to quit taking the new therapy. Many patients took antihistamines or topical steroids to manage the rash effect, but there wasn’t really data to assess how well that worked, the agency said. Researchers still don’t know why the drug is associated with rash.
Anemia, a condition which causes weakness and fatigue, was found in 36 percent of patients on the Veretx drug in tandem with the standard meds, while that effect was seen in just 15 percent of patients on the two standard treatments alone, the FDA said. The effect was mostly managed through reducing the dose of one of the standard medications—ribavirin—the agency said. Researchers also said patients on the new drug had a higher incidence of rectal side effects, like hemorrhoids and inflammation. Patients also experienced fatigue, nausea, headache, and diarrhea at higher rates on the Vertex drug—all pretty common stuff for many new drugs.
The FDA staff’s conclusion was about as kind as these documents get. It said Vertex submitted “substantial” evidence for its drug’s toxicology, clinical pharmacology, clinical virology, and clinical trial data. “We look forward to an interactive and productive discussion of the data,” FDA staff said.
The agency plans to webcast the public advisory committee on Thursday, one day after it completes a hearing for a rival hepatitis C drug from Merck. You can check the briefing document relevant to Vertex here, the agenda, and see who’s on the committee.
