Pharmacyclics, J&J Get FDA Nod for Chronic Lymphocytic Leukemia Drug

2/12/14Follow @xconomy

One of the important new medicines for blood cancer just got approved by the FDA for a large new group of patients.

Sunnyvale, CA-based Pharmacyclics (NASDAQ: PCYC) and its partner Johnson & Johnson (NYSE: JNJ) won clearance today from the FDA to start marketing ibrutinib (Imbruvica) as a new therapy for patients with chronic lymphocytic leukemia who have gotten at least one other treatment. The FDA nod opens up a big new potential market, as more than 15,000 patients in the U.S. are diagnosed with chronic lymphocytic leukemia each year, according to National Cancer Institute statistics cited by the FDA.

The drug, designed to fight tumors by binding with a molecular target called Bruton’s tyrosine kinase, is considered by many doctors to be one of the more important cancer treatments to come along in years. It was first approved by the FDA in November as a treatment for a relatively small group of patients with mantle cell lymphoma. Like many cancer drugs, its initial price is sky-high—about $10,930 for mantle cell lymphoma patients who get a 30-day supply of the recommended dose. That works out to about $131,000 a year. This new approval for chronic lymphocytic leukemia is for a 420 milligram once-daily dose, which works out to a lower price of approximately $8,200 per month, or $98,400 per year, according to a spokesman for Pharmacyclics.

That sort of pricing, multiplied by the growing numbers of eligible patients with two types of cancer, has prompted analysts to forecast peak annual sales of more than $6 billion.

“Today’s approval provides an important new treatment option for [chronic lymphocytic leukemia] patients whose cancer has progressed despite having undergone previous therapy,” said Richard Pazdur, director of the FDA’s cancer drug review office, in a statement. “The FDA completed its review of Imbruvica’s new indication under the agency’s accelerated approval process, which played a vital role in rapidly making this new therapy available to those who need it most.”

Nobody can say for sure whether the new drug is effective at helping chronic lymphocytic leukemia patients live longer. Today’s FDA approval was based on a study of 48 patients who had been diagnosed an average of 6.7 years earlier, and had gotten an average of four previous therapies for chronic lymphocytic leukemia. Despite being that sick, about 58 percent of patients saw their tumors shrink after getting the new drug, and the responses lasted between 5.6 months and 24.2 months, according to today’s FDA statement. The most common side effects were a depletion of clot-forming platelet cells in the blood, bruising, diarrhea, and depletion of white and red blood cells.

The drug’s prescribing information also includes a warning about the possibility of hemorrhage, infections, kidney toxicity, and secondary malignancies. Five of the 48 patients dropped out of the study because of side effects, Pharmacyclics said in a statement.

By winning approval in chronic lymphocytic leukemia, the new drug will compete with other drugs on the market like Roche/Genentech’s obinutuzumab (Gazyva), and others nearing the market, such as Gilead Sciences’ idelalisib.

Pharmacyclics is planning to hold a conference call with analysts at 2:30 pm ET/11:30 am PT today to discuss the new approval.

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