Hepatitis C drug from Vertex sails through test
Advisers to FDA endorse telaprevir
SILVER SPRING, Md. — Vertex Pharmaceuticals Inc. yesterday cleared the toughest hurdle in its quest for US approval of what is expected to be the first blockbuster drug launched by a Massachusetts company in nearly a decade.
A medical advisory committee unanimously recommended that telaprevir, the experimental drug Vertex developed to treat the liver-ravaging hepatitis C virus, be approved by the Food and Drug Administration. The agency, which typically follows the panel’s advice, is scheduled to make a decision May 23.
“We’re absolutely thrilled,’’ said Dr. Shelley George, vice president in the hepatitis C therapeutic area for Vertex. “This is a fantastic milestone for the patients and for the company.’’
The Cambridge biotech has been working on the treatment since 1993. Once approved, telaprevir would be given a new brand name and become the first drug commercialized solely by Vertex in its 22-year history. The company is gearing up to compete with a rival tablet from Merck & Co. in a market that an alysts project will initially top $2 billion a year, and could grow to $10 billion or more.
Blockbuster drugs are those that ring up more than $1 billion in annual revenue. The last one brought to market by a Massachusetts company was Tysabri, a multiple sclerosis treatment from Biogen Idec Inc. of Weston.
Tysabri first won FDA approval in 2004, but was later pulled from the market because of safety concerns. It was relaunched in 2006.
The antiviral drugs advisory committee, an independent panel of medical authorities from across the country, voted 18 to 0 to recommend approval of telaprevir. The drug will be prescribed in combination with two other drugs currently on the market: pegylated interferon and ribavirin.
After a daylong hearing packed with testimony from Vertex executives, regulators, doctors, and patients, who spoke of telaprevir’s effectiveness but also warned of side effects from rashes to anemia, the panel concluded the drug’s risks were manageable.
“The benefits far outweigh the risks,’’ said Dr. Michael E. Bigby, an advisory committee member and dermatologist at Beth Israel Deaconess Medical Center in Boston.
Hepatitis C patient Kelly Ann Hester, a billing supervisor for a Pennsylvania health care provider, told the committee she was cured by telaprevir in a clinical trial after multiple rounds of treatment with current drugs failed. “I’ve been through every treatment that’s been made available in the past 18 years,’’ she said.
“I was in a place where I was living to die,’’ she said. “Now I’m living until I die, which is a whole new concept for me.’’
Medical experts estimate that as many as 3.9 million Americans, three quarters of whom don’t know it, are infected by the hepatitis C virus. Many baby boomers contracted the disease through drug use or blood transfusions decades ago. If untreated, the disease can lead to liver scarring or liver cancer and is ultimately fatal.
Current drugs cure fewer than half the patients with the hardest-to-treat form of the disease, but telaprevir cured 79 percent of these first-time patients in clinical trials — in half the time as today’s drugs. Like its Merck rival, boceprevir, telaprevir acts by blocking the protease enzyme that allows the hepatitis C virus to replicate.
“It’s a stunning achievement that we will be able to cure nearly 80 percent of [first-time] patients and nearly as many relapsers,’’ said another advisory committee member, Dr. Lawrence S. Friedman, chairman of the medicine department at Newton-Wellesley Hospital in Massachusetts.
Trading in Vertex shares was halted on the Nasdaq Stock Market yesterday. Exchange officials sometimes close access to a drug company’s stock temporarily to prevent speculative trading if a medication critical to the company comes before an FDA panel.
While yesterday’s vote was unanimous, committee members grilled Vertex executives on everything from what steps they would take to educate patients about side effects to why only a small number of African-Americans took part in clinical trials.
“I was disappointed your recruitment of blacks was so low given the significant portion of the disease in that population,’’ said Dr. Elizabeth Connick, an associate professor of medicine at the University of Colorado Denver.
Vertex executives said they are setting up a 24-hour telaprevir telephone hotline to educate patients and doctors. They also are considering a post-approval trial aimed at African-Americans, for whom cure rates are lower than the general population.
The advisory committee’s endorsement of telaprevir came one day after it recommended approval of boceprevir, which Merck, based in Whitehouse Station, N.J., will market under the name Victrelis. Merck has said it expects a final FDA decision next month but has not specified a date.
Analysts have estimated that the Vertex drug could capture more than two thirds of the market. But Merck, one of world’s largest pharmaceutical companies, still has commercial advantages — a well-established sales force and a long history of selling antiviral drugs, including the two existing hepatitis C treatments.
The Vertex and Merck drugs have not been compared head to head. But advisory panel members said they both represent a leap forward.
“I think this is a long-awaited landmark day for patients with hepatitis C,’’ said Dr. Barbara H. McGovern, associate professor at Tufts University School of Medicine in Boston.
Robert Weisman can be reached at firstname.lastname@example.org.