Hamburg provides update on breakthrough, biosimilars pathways

In a Kalydeco ivacaftor from Vertex Pharmaceuticals Inc. (NASDAQ:VRTX) as monotherapy and in combination with Vertex's VX-809; ibrutinib from Pharmacyclics Inc. (NASDAQ:PCYC) to treat relapsed or refractory mantle cell lymphoma (MCL) and Waldenstrom's macroglobulinemia; and LDK378 from Novartis AG (NYSE:NVS; SIX:NOVN) to treat anaplastic lymphoma kinase ( ALK)-positive metastatic non-small cell lung cancer (NSCLC).

Breakthrough drug designation, which was created by the FDA Safety and Innovation Act, commits FDA to collaborate with a sponsor to enable expedited development and review of compounds for serious or life-threatening diseases that show substantial improvements over existing treatments in early trials. Hamburg said FDA is developing guidance to describe and explain the criteria for its expedited development and review programs, including breakthrough designation (see BioCentury, Dec. 3, 2012). ...