Novartis is the latest pharma company to have a drug classed as a “breakthrough therapy” by the FDA, accelerating its passage through development.
The company said the FDA has given the status to its LDK378 drug candidate for metastatic non-small cell lung cancer (NSCLC) patients who test positive for a genetic mutation known as anaplastic lymphoma kinase (ALK) and are unable to be treated with Pfizer’s recently-launched Xalkori (crizotinib).
The status was created by the FDA Safety & Innovation Act (FDASIA) and is intended to expedite the development and review of drugs that show substantial improvement over existing therapies for serious and unmet medical needs.
It works in a similar way to the agency’s long-established fast-track programme for accelerated approval and priority review of important new medicines, and also establishes a higher level of FDA guidance. It includes measures such as reducing the number of patients who need to be enrolled into placebo-controlled trials and shortening their duration.
“This breakthrough therapy designation will allow us to collaborate more closely with the FDA and potentially to expedite the availability of an important new treatment option for patients with ALK+ NSCLC,” commented Alessandro Riva, head of oncology development at Novartis.
The first published FDA breakthrough therapy designations were awarded to Vertex Pharma for its cystic fibrosis candidates Kalydeco (ivacaftor) and the combination of Kalydeco and VX-809.
Novartis said phase I trials with LDK378 with a range of ALK-positive cancers showed a combined complete response, partial response and unconfirmed partial response of 80 per cent among patients whose disease had progressed despite crizotinib treatment.
The company has now started two phase II clinical trials of LDK378 in ALK-positive NSCLC patients, and aims to start pivotal studies later this year with marketing applications expected in 2014.