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Advanced Hepatitis Drug Nears FDA Approval

Vertex Pharmaceuticals Inc., maker of the HIV treatment Lexiva, has made a business of tackling some of the world’s most difficult-to-treat diseases.

Next year, the company plans to submit a first-of-its-kind drug candidate to the FDA for treating chronic hepatitis C virus infections in people who have failed standard treatments.

Analysts see the drug, called telaprevir, as a potential blockbuster because roughly 650,000 of the 3.2 million Americans living with HCV infections have failed standard therapies.

Four times more prevalent than HIV in the United States, the deadly liver disease also represents a major concern among aging baby boomers, who represent more than half of the patients in the U.S.

A report commissioned by Vertex this year demonstrates how medical costs for HCV patients could rise from $30 million today to $85 billion in the next 20 years. Government payers like Medicare will increasingly be footing the bill on treatment costs, according to Vertex CEO Matthew Emmens.

“That is a tsunami that is making its way toward the health care industry,” Emmens said during a recent stopover visit to the company’s San Diego research laboratories.

Emmens took over as chairman and CEO in May following a career with Britain’s Shire Pharmaceuticals Group plc, maker of Adderall, where he succeeded Vertex founder Joshua Boger as CEO. The two have known each other for years, dating back to their early days at Merck & Co. Inc. in the 1980s.

Roughly 175 researchers work at the La Jolla facility, formerly home to Aurora Pharmaceuticals before Vertex acquired the company for $600 million in stock in 2001. Most of its scientists are focused on an experimental cystic fibrosis drug candidate, known as VX-770, which could reach the FDA following telaprevir.

Before it does, Vertex could see its first commercial revenue stream if telaprevir passes regulatory muster. For the three months ended Sept. 30, Vertex reported a net loss of $149.6 million on revenues of $25 million.

Promising Data Released

Vertex wowed investors earlier this month when the company released some preliminary data on the drug at a Boston conference of the American Association for the Study of Liver Diseases.

In 161 patients treated for the first time, the drug produced a clinical cure, defined as a sustained virological response, in 80 percent of those taking the drug. It had the same effect when given twice a day or three times a day.

Earlier results involving a small number of patients who had failed to respond to standard treatments or relapsed after taking the standard drugs, achieved 57 percent and 90 percent clinical cure rates, respectively, when the drug was added to their regimen.

In some of the patients, the virus was undetectable in their bloodstream after 24 weeks.

“Here, you have a really dramatic improvement because you’re cutting the duration of treatment for a patient by half but you also have improved cure rate,” said Leerink Swann LLC analyst Howard Liang.

Eight of the 117 patients enrolled in the earlier study dropped out due to adverse side effects, including four who dropped out because of rash, and one who cited anemia.

Physicians eager for a new drug have been “warehousing” patients in anticipation, according to Liang.

“It has happened before that when there’s a new product coming, patients and doctors will wait for a new treatment,” he said. “Because the disease is a chronic disease, it is possible to wait.”

Preparing For FDA Submission

Vertex is running pivotal Phase 3 trials that it hopes will clear the way for an FDA submission in the second half of next year. It faces competition from some major drug makers, including Merck & Co., which is testing an experimental drug called boceprevir in Phase 3 trials.

Vertex is advocating for earlier screening of HCV, something that could draw additional patients its way.

The issue has also caught the attention of Washington lawmakers. A bill making its way through the House Committee on Energy and Commerce, known as H.R. 3974, would add hepatitis education and prevention programs directed by the Secretary of Health and Human Services.


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