Novartis recently announced that the FDA has granted “breakthrough therapy” status to LDK378 for the treatment of ALK-positive, crizotinib (Xalkori) therapy-naïve patients with metastatic non-small cell lung cancer (NSCLC). The “breakthrough therapy” status is derived from the FDA Safety and Innovation Act (FDASIA) and is intended to expedite the development and review of certain new drugs that offer a significant improvement over existing treatments for critical and unmet medical needs. The approval process for “breakthrough therapy” eligible drugs is similar to the FDA’s long-established accelerated approval fast track process and priority review for priority new drugs. ”Breakthrough therapy status allows us to work more closely with the FDA to accelerate the adoption of new drugs,” commented Alessandro Riva, head of oncology drug development at Novartis. The first FDA-approved “breakthrough therapy” status was granted to two of Vertex Pharmaceuticals’ drugs, Kalydeco (ivacaftor) and a combination of Kalydeco and VX-809. Novartis claims that 80% of patients with progressive disease after treatment with crizotinib in a series of Phase I clinical trials in ALK+ cancers showed responses to LDK378, including complete responses, partial responses and unconfirmed partial responses. Novartis has now initiated two Phase II clinical trials of LDK378 in patients with ALK+ NSCLC with the aim of initiating pivotal studies later this year to enable the submission of a marketing application for LDK378 in 2014.