J&J-Pharmacyclics Breakthrough Drug Wins U.S. Approval

Johnson & Johnson and Pharmacyclics Inc. won U.S. approval for their breakthrough therapy to treat a rare blood cancer, becoming the second drug to gain marketing clearance under the process created to speed new medicines to seriously ill patients.

The Food and Drug Administration said today it cleared ibrutinib, to be known as Imbruvica, for previously treated patients who have mantle cell lymphoma. The FDA had granted the medicine breakthrough status, which means preliminary evidence indicated it may offer substantial improvement over current treatment, the agency said in a statement.

Imbruvica is more tolerable than traditional combination medications for cancers that can cause serious side effects such as low red cell counts requiring blood transfusions. Peak worldwide sales for the pill may reach $6.5 billion in 2026 if it’s approved for more cancers, with $3 billion from the U.S., Michael Yee an analyst with RBC Capital Markets, said in a note to clients.

“Often people are as much in fear of the treatment as they are the disease itself,” Chief Executive Officer Robert Duggan of Sunnyvale, California-based Pharmacyclics said in an interview before the FDA’s announcement. “We feel in this new era the treatment will be much friendlier.”

Pharmacyclics rose 3.5 percent to $123.82 at the close in New York. The shares have more than doubled this year. J&J declined less than 1 percent to $93.34.

Aggressive Cancer

Mantle cell lymphoma is an aggressive blood cancer that starts in the white blood cells and accounts for about 6 percent of all non-Hodgkin lymphoma cases in the U.S., the FDA said. The disease affects fewer than 200,000 people in the U.S. with about 2,900 new cases diagnosed each year, New Brunswick, New Jersey-based J&J said in a statement. By the time it is diagnosed, the cancer usually has spread to the lymph nodes, bone marrow and other organs.

“Imbruvica’s approval demonstrates the FDA’s commitment to making treatments available to patients with rare diseases,” said Richard Pazdur, director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research. “The agency worked cooperatively with the companies to expedite the drug’s development, review and approval, reflecting the promise of the breakthrough therapy designation program.”

Competing Drugs

Imbruvica is the third drug approved for patients with mantle cell lymphoma after Velcade, from J&J and Takeda Pharmaceutical Co., and Celgene Corp.’s best-selling Revlimid, the agency said. The medicine is the first in a class of drugs called Burton’s tyrosine kinase inhibitors that block the BTK enzyme from promoting proliferation and survival of malignant cells.

The companies also sought approval for the medicine in chronic lymphocytic leukemia and small lymphocytic lymphoma.

“Breakthrough therapy designation has been quite powerful for us,” Duggan said. “It has given us almost instant access to key players in the FDA.”

The drug was approved based on mid-stage trials, while final trials will produce results as early as the first half of 2014 in CLL and later in the same year or in 2015 on mantle cell lymphoma, Maria Fardis, chief of oncology operations for Pharmacyclics, said in an interview.

Pharmacyclics shares marketing responsibilities in the U.S. with J&J, the world’s biggest seller of health-care products. The companies shared the cost of development and worldwide will equally split profits.

Pharmacyclics said in July it would be eligible for a $350 million payment from J&J upon approval.