Imbruvica, Drug to Treat Blood Cancer, Gains F.D.A. Approval

A new cancer drug that has attracted considerable attention from doctors and investors won approval from the Food and Drug Administration on Wednesday.

The drug, which will be called Imbruvica, was approved as a treatment for relapses of a rare blood cancer called mantle cell lymphoma. But the companies that developed it, Pharmacyclics and Johnson & Johnson, have also applied for approval to treat a more common cancer, chronic lymphocytic leukemia, or C.L.L.

"In two diseases that are very hard to treat, it induces durable remissions," Dr. John C. Byrd, director of hematology at the Ohio State University Comprehensive Cancer Center, said in an interview. "This is really beating the pants off anything we have right now."

Some Wall Street analysts have projected sales of several billion dollars a year for Imbruvica, which is known generically as ibrutinib.

Shares of Pharmacyclics, based in Sunnyvale, Calif., jumped $4.15, or 3.5 percent, to close at $123.82 on Wednesday, giving the company a market value of $9.1 billion. The stock of the 22-year-old company, which has not gotten a drug to market until now, was trading for less than $1 in 2008, shortly before clinical trials of ibrutinib began. It is now up more than 140 percent over the last year.

Imbruvica will cost about $91 a pill, with four pills taken together once a day. That works out to about $10,900 a month, or more than $130,000 a year, in line with the recent trend of ever-increasing cancer drug prices.

Robert W. Duggan, chief executive of Pharmacyclics, said the price reflected that "mantle cell is an orphan disease" and that the two companies had spent close to $1 billion to bring Imbruvica to market. He said that for C.L.L., which requires fewer pills, the price would be $8,200 a month. The companies will offer financial assistance to many patients.

The F.D.A. had designated Imbruvica a "breakthrough therapy" for three types of cancer under a program established by Congress last year. That designation contributed to the drug's approval more than three months before the F.D.A.'s deadline in late February.

Mantle cell lymphoma accounts for about 6 percent of cases of non-Hodgkin's lymphoma, according to the F.D.A. There are about 11,000 people with the disease in the United States and nearly 3,000 new cases a year, according to Pharmacyclics.

Celgene's Revlimid and Takeda's Velcade are also approved to treat the disease. Chemotherapy and Genentech's Rituxan are also used.

Imbruvica was approved based on a clinical trial testing it in 111 patients whose cancer had recurred after at least one prior therapy. The cancer shrank in about 49 percent of patients and disappeared in 17 percent. These responses lasted a median of 17.5 months. It is not yet clear if the drug prolongs lives.

Side effects included diarrhea, infections and bleeding or bruising and decreases in platelets and infection-fighting white blood cells.

There was some concern among investors that the drug was not approved for C.L.L. on Wednesday. Executives at Pharmacyclics and Johnson & Johnson said the F.D.A. was continuing to evaluate that application.

Imbruvica is the first approved drug that works by inhibiting Bruton's tyrosine kinase, a protein that contributes to the proliferation and survival of B cells, which are the white blood cells that turn malignant in mantle cell lymphoma.