Vertex Seeks FDA Green Light for Hepatitis C Drug; Chomps at the Bit For Fast Review

Xconomy Boston — 

Vertex Pharmaceuticals has spent 15 years working to reinvent the way hepatitis C infection is treated, and now the fruits of that labor are in the hands of the U.S. Food and Drug Administration.

Cambridge, MA-based Vertex (NASDAQ: VRTX), which has West Coast operations in San Diego, is announcing today that it has filed its new drug application, in which it is seeking clearance from the FDA to start selling telaprevir as a hepatitis C treatment in the U.S. The company is seeking a faster-than-usual six-month regulatory review, instead of the usual 10 month period, a request the FDA sometimes grants for potentially lifesaving or groundbreaking new therapies. The filing comes right on schedule, as the company said throughout this year it planned to complete its application before year’s end.

The company’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 drug, in addition to standard meds, was able to essentially cure three-fourths of patients getting their initial round of therapy for hepatitis C infections. The second study showed that the drug could shorten the standard treatment time by half. A third trial proved the new treatment could cure two-thirds of patients who failed to respond to a prior round of therapy. Taken together, Vertex is hoping the body of evidence in this application will pave the way for a completely new treatment strategy that will roughly double the cure rate of the existing regimen, while requiring patients to put up with fewer of the nasty flu-like symptoms that dog the current standard of care.

An estimated 6 million patients in the U.S. and Europe have hepatitis C, out of an estimated 170 million worldwide. While many go untreated today, Vertex and its investors are hoping that if the new treatment is approved, it will open the floodgates for patients to rid themselves of the infection that can cause significant liver damage and death later in life. If Vertex can get the FDA’s green light, it will be the first major product for this company founded in 1989, which has accumulated a deficit of $3.26 billion through the end of September. If Vertex wins market clearance in 2011, as most analysts expect it will, the payoff could come relatively quickly. U.S. sales alone could amount to more than $2 billion after a couple years because of the pent-up demand built for the drug during the clinical trial process, analysts say. Vertex’s market valuation yesterday was about $7 billion, based mostly on the projected value of this new treatment for hepatitis C.

“This submission is a milestone in our more than 15-year effort to change the way hepatitis C is treated,” said Matt Emmens, Vertex’s CEO, in a statement. “We are committed to working closely with the FDA to make telaprevir available as quickly as possible to the millions of people with hepatitis C who need new medicines to increase their chances for a viral cure.”

Hepatitis C is currently treated with pegylated interferon alpha and ribavirin, a combination therapy that causes flu-like symptoms, and which many patients consider worse than the disease itself, which can lie dormant for years. Telaprevir, an oral pill taken three times a day, is designed to attack the virus through a different mechanism than the other meds, as a member of the class of protease inhibitors. Vertex’s success at boosting viral cure rates and reducing the dependence on a widely loathed standard regimen has invited one serious late-stage competitor—Merck’s boceprevir. It has also helped inspire a wide array of other companies to attack the virus in various cocktail regimens with nucleoside inhibitors and non-nucleoside inhibitors, through a strategy that resembles the multi-drug approaches that are now standard therapy for HIV.

There are a lot of details about the safety and effectiveness of this drug in its main clinical trials, which I won’t attempt to repeat here. You can follow the links to our past coverage, including a Phase III results preview story in May, followed by the reports on the critical trials known as Advance, Illuminate, and Realize. You can also get a sense of Vertex’s vision for commercializing this drug through a profile of Emmens that ran back in January.

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2 responses to “Vertex Seeks FDA Green Light for Hepatitis C Drug; Chomps at the Bit For Fast Review”

  1. I have 1st stages of scirosis of liver will i be able to take telepravir?, for my gi dr. says i cannot take current treatment{ combo drugs} for it would be lifethreatening.