FDA advisory panel gives thumbs up to Vertex's Telaprevir...

FDA backs Vertex hepatitis drug efficacy, shares jump
Tue, Apr 26 2011

By Lisa Richwine

WASHINGTON (Reuters) - A hepatitis C drug from Vertex Pharmaceuticals Inc cured more patients than previously reported, U.S. health reviewers said, boosting the approval chances for a pill expected to transform treatment of the liver-destroying disease that afflicts millions.

Vertex shares rose 12 percent to their highest level in a decade after comments from Food and Drug Administration staff on Tuesday that analysts said favored the medicine's approval.

Shares of Merck, whose rival hepatitis C treatment also faces an FDA panel's review this week, rose 2.2 percent.

Both medicines are expected to reap more than $1 billion in sales if approved after demonstrating an ability to cure far more patients than current standard drugs, in some cases in half the time.

Vertex's telaprevir eliminated the hepatitis C virus in 79 percent of newly treated patients, FDA reviewers said. That was higher than the 75 percent Vertex reported earlier because the FDA used a different cure rate calculation.

Analysts said the higher effectiveness, plus few FDA safety concerns, meant an advisory panel would likely recommend approval of the drug on Thursday.

"We now expect telaprevir to get a relatively clean recommendation (and hence approval) with no significant exclusions, restrictions, warnings or caveats," Sanford Bernstein analyst Geoffrey Porges said in a research note.

Current therapies require almost a year of treatment and often cause flu-like symptoms that are tough to tolerate, with only about a 40 percent success rate. Both of the new medicines work by blocking a protein called protease that the virus needs to replicate.

WAITING FOR TREATMENT

Doctors say tens of thousands of patients have been delaying treatment in anticipation of the new anti-virals, which still must be taken in combination with the older drugs ribavirin and interferon.

Hepatitis C is a bloodborne liver disease that can lead to chronic liver problems, liver cancer, cirrhosis and death. It is spread mainly through reuse of needles such as those used for illegal drugs and tattoos, or through blood transfusions before 1992 when screening began. About 3.2 million Americans and 170 million people worldwide have the disease.

Analysts widely expect the Vertex pill to dominate the market with its greater effectiveness. Telaprevir's 79 percent success rate compares with up to 66 percent for Merck's drug, boceprevir, in previously untreated patients. It also had a higher cure rate than the Merck drug in patients who had failed to be helped by prior therapy.

"Overall, the FDA review team's independent analyses confirmed the applicant's primary efficacy findings," FDA staff said in a summary prepared for the advisory panel of outside experts.

The FDA reviewers said they would ask the panel to discuss rashes and anemia seen in clinical trials of telaprevir. Those issues were known and analysts do not expect them to derail the drug's approval.

An FDA panel will review the Merck drug on Wednesday. In documents released on Monday, agency reviewers said Merck's drug appeared effective but highlighted anemia and other safety issues for panel discussion.

The FDA usually approves medicines that win advisory panel backing. An approval decision for either drug could come as early as May.

(Additional reporting by Toni Clarke and Bill Berkrot, editing by Michele Gershberg, Gerald E. McCormick, Derek Caney and Bernard Orr)