Novartis says that regulators in the USA have agreed to fast-track a treatment developed by the Swiss major for a rare type of lung cancer.
The compound, known as LDK378, has received 'breakthrough therapy' designation from the US Food and Drug Administration for the treatment of patients with anaplastic lymphoma kinase positive (ALK+) metastatic non-small cell lung cancer (NSCLC) who had progressed during treatment with, or were intolerant to, Pfizer's Xalkori (crizotinib). Novartis notes that there are limited treatment options for patients with ALK+ NSCLC, who tend to be non-smokers and younger than patients without an ALK translocation.
According to the FDA, 'breakthrough therapy' designation is granted to drugs that treat serious or life-threatening conditions if it has "demonstrated substantial improvement over an available therapy on at least one clinically significant endpoint". The designation includes "all of the fas-track programme features, as well as more intensive FDA guidance" and is different to accelerated approval and priority review, which can also be granted if relevant criteria are met.
In an 88-patient Phase I study, a response rate of 80% was observed in those on LDK378 who had experienced disease progression after treatment with Xalkori. Novartis has initiated two Phase II studies and will begin several Phase III trials later this year. First regulatory filing is anticipated by early 2014.
Alessandro Riva, head of oncology development and medical affairs at Novartis, said LDK378 is "a strong example of our research approach, which focuses on identifying the underlying cause of disease pathways". He added that getting 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".
The first breakthrough therapy designations were granted by the FDA at the beginning of this year to Vertex Pharmaceuticals' Kalydeco (ivacaftor) for potential additional indications beyond the drug's currently-approved use in cystic fibrosis in patients who have the G551D mutation and for a combination of Kalydeco with the investigational compound VX-809.