US FDA grants "breakthrough" status for Janssen and Pharmacyclics ibrutinib

US health care giant Johnson & Johnson (NYSE: JNJ) subsidiary Janssen Research & Development, says that the US Food and Drug Administration has granted Breakthrough Therapy Designations for the investigational oral agent ibrutinib as a monotherapy for two B-cell malignancies: in patients with relapsed or refractory mantle cell lymphoma (MCL) who have received prior therapy, and in patients with Waldenstrom's macroglobulinemia (WM).

More than a year ago, Janssen executed an agreement with fellow USA-based Pharmacyclics (Nasdaq: PCYC) to jointly develop and market the anti-cancer compound, previously dubbed PCI-32765 (The Pharma Letter December 9, 2011). Under the terms of the deal, Pharmacyclics received an upfront $150 million. The company will also get up to an additional $825 million in development and regulatory milestone payments, based upon continued development, regulatory progress and approval of the product, for total potential upfront and milestone payments of $975 million. Two $50 million milestone have already been earned by Pharmacyclics.

Enacted as part of the 2012 FDA Safety and Innovation Act (FDASIA), Breakthrough Therapy Designation is intended to expedite the development and review time for a potential new medicine "to treat a serious or life-threatening disease or condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints, such as substantial treatment effects observed early in clinical development."