Novartis lung cancer drug earns breakthrough therapy designation from FDA
by Jeffrey Bouley  |  Email the author


BASEL, Switzerland—Novartis AG announced March 15 that its investigational compound LDK378 had received breakthrough therapy designation by the U.S. Food and Drug Administration (FDA) 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, crizotinib. Crizotinib is marketed by Pfizer under the brand name Xalkori.  
The designation is important—and appropriate—Novartis notes, as there exist very limited treatment options for patients with ALK+ NSCLC, who tend to be non-smokers and younger than NSCLC patients without an ALK translocation.  
The FDA has indicated that the breakthrough therapy designation is intended to expedite the development and review of drugs that treat serious or life-threatening conditions if the therapy has demonstrated substantial improvement over an available therapy on at least one clinically significant endpoint. This designation includes all of the features of FDA's fast-track program, with the addition of more intensive FDA guidance. The Breakthrough Therapy designation is considered a distinct status from both accelerated approval and priority review, which can also be granted to the same drug if relevant criteria are met, Novartis explains.  
"LDK378 is a strong example of our research approach, which focuses on identifying the underlying cause of disease pathways," said Alessandro Riva, global head of oncology development and  medical affairs at Novartis Oncology. "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."  
Initial results from a Phase I study investigating the maximum tolerated dose, safety, pharmacokinetics and anti-tumor activity of LDK378 in 88 patients with ALK+ advanced malignancies and who had progressed during treatment with, or were intolerant to, crizotinib, showed marked responses in a majority of patients. According to Novartis, a response rate (including complete response, partial response and unconfirmed partial response) of 80 percent was observed in the patients who had experienced disease progression after crizotinib treatment.  
Two Phase II clinical trials are already underway to further evaluate the compound in this patient population, with plans to begin several Phase III clinical trials later this year. First regulatory filing is anticipated by early 2014. LDK378 is one of the at least 14 blockbuster drugs Novartis said in November 2012 that it intends to bring to market by 2017.
NSCLC is the most common type of lung cancer, accounting for as much as 90 percent of all lung cancer cases. Somewhere between 3 percent and 8 percent of patients with NSCLC have the ALK gene mutation.   
Code: E03151301

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