Pharmacyclics Gains ‘Breakthrough’ Status for Cancer Drug

Pharmacyclics Inc., a drugmaker developing an experimental therapy for blood cancers, won “breakthrough” status from U.S. regulators for the medicine that may lead to quicker marketing approval.

Pharmacyclics and its partner, New Brunswick, New Jersey-based Johnson & Johnson, will submit the therapy, ibrutinib, to the Food and Drug Administration for approval before the end of the year, the companies said today in separate statements.

“This is a historic moment in oncology,” Bob Duggan, chief executive officer of Sunnyvale, California-based Pharmacyclics said in the statement. “We are truly honored to have received this breakthrough designation and are pleased for patients and clinicians with the FDA’s decision to expedite the development of ibrutinib.”

The breakthrough designation was created in legislation passed last year reauthorizing the FDA’s user fee programs for drug and device reviews. Companies that receive the status will have closer communication with top FDA staff to move drugs for serious diseases to market more quickly, Janet Woodcock, director of the agency’s Center for Drug Evaluation and Research, said yesterday in an interview.

New drugs generally need three phases of clinical testing on safety and effectiveness to win FDA approval. Woodcock said the breakthrough status may lead to approval after just an expanded early stage clinical trial.

Ibrutinib blocks an enzyme called Bruton’s tyrosine kinase that aids certain cancers in spreading. The drug has breakthrough status for patients with Waldenstrom’s macroglobulinemia, and mantle cell lymphoma patients that have failed other therapies. Pharmacyclics said it’s currently testing the drug in five late-stage trials.

Mantle cell lymphoma is a type of B-cell non-Hodgkin lymphoma that typically strikes older adults, with about 5,000 new cases each year, Pharmacyclics said. Waldenstrom’s macroglobulinemia is a rarer type of lymphoma that affects 1,500 people a year and there are no approved treatments.