Vertex Pharmaceuticals is heading down the home stretch in its bid to transform treatment of hepatitis C, and this week the public will get an up-close look at the risks and benefits of its drug that could change the standard of care for millions of people.
Cambridge, MA-based Vertex (NASDAQ: VRTX) will have its big moment on Thursday, when it appears before the FDA’s advisory panel of antiviral drug experts in Silver Spring, MD. The committee will review Vertex’s application to start selling a thrice-daily pill, telaprevir, as a new therapy for hepatitis C in the U.S. The same group will scrutinize boceprevir, a rival compound from New Jersey-based Merck (NYSE: MRK), one day earlier. FDA staff is also expected to release briefing documents on the drugs this week. The agency, after weighing input from staff and the panel, has until May 23 to decide whether to clear the Vertex drug for sale.
Analysts expect both Vertex and Merck to win recommendations from the FDA advisory panels, but the hearings are important because all drugs have risks, and questions from the experts could shed light on how well the new drugs will be embraced in the market. While Merck is a diversified company with lots of products, the stakes could hardly be any higher for Vertex, which reported a net loss of $754 million last year during its last big pre-market push for telaprevir. If it’s FDA approved, many analysts predict the Vertex drug will surpass $2 billion in U.S. sales within a couple years, offering the company a chance to start recouping 16 long years of investment in R&D. If Merck and Vertex win approval, they will both be offering much higher chances of a cure than ever before to 3 million people in the U.S. with the chronic liver infection, which can lead over time to cirrhosis and death.
“This is a very exciting time for patients, and we feel it’s important that patients have new options for treatment,” says Shelley George, Vertex’s vice president of medical affairs, and hepatitis C therapeutic area lead, who will deliver a presentation at Thursday’s panel. “We’re very excited to present our data, and we’re confident.”
Head-spinning amounts of information about this drug’s safety and effectiveness profile have already been made public, through press releases and medical meetings over the years. But for those checking in on this week’s proceedings, here is a quick recap of what we know:
Vertex’s application is based primarily on a trio of Phase III clinical trials that enrolled more than 2,000 patients. One trial showed that the new Vertex drug, given in addition to standard pegylated interferon alpha and ribavirin, was able to essentially cure three-fourths of patients getting their first course of therapy for hepatitis C infections. The second study showed that the new drug could shorten the standard treatment time by half—bringing a 48-week regimen down to 24 weeks. A third trial proved the new treatment could cure two-thirds of patients who failed to respond to a prior round of therapy.
Most patients choose not to seek treatment today because they have to endure almost a year of flu-like symptoms with a less than 50-50 shot of getting cured. Adding telaprevir turns the equation around completely, by offering a 75 percent shot of a cure, while requiring patients to put up with the flu-like side effects of the standard drugs for less than six months.
The panel could spend some time asking detailed questions about the side effects of this new combo regimen, so I made sure to ask George about that during a short call last week. She noted that more than 40 different studies that include about 4,000 patients make up Vertex’s application to the FDA. “It’s an extremely well-studied medicine,” George says.
The most common side effects among patients on telaprevir were rash and anemia, George says. As many as two-thirds of patients on the Vertex drug reported a rash in studies, although George says that 90 percent of those cases were mild-to-moderate in severity. Through experience in trials, Vertex and physicians learned to manage the rash by using immune-suppressing corticosteroid drugs or antihistamines, or by taking patients off telaprevir when it was no longer necessary, George says. About 1 percent of patients dropped out of the studies because of the rash, and the dropout rate declined as Vertex learned how to better manage the side effect, she says.
About one-third of patients on the current standard drugs get anemia, a depletion of oxygen-carrying red blood cells that makes people feel weakness and fatigue. The rates of anemia climbed slightly when researchers added the Vertex drug to the mix, George says. Most of the anemia cases in Vertex’s pivotal studies were managed the usual way, she says, as doctors lowered the dose of one of the standard drugs—ribavirin.
While Vertex and Merck have never studied their respective compounds head-to-head, it’s inevitable that the panel, doctors, and investors will make their judgments on which one they prefer as they appear on track to reach the market in what you could call a dead-heat. Regardless of which gets to the market first, most analysts see Vertex’s drug as best-in-class. We’ll have a clearer answer to that question after hearing what the FDA staff has to say this week about the drugs in its briefing documents, and in the questions that the FDA panel chooses to raise.
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