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Novel Compound LDK378 Receives Breakthrough Designation for ALK+ NSCLC

Ben Leach
Published: Friday, Mar 15, 2013

Alessandro Riva

Alessandro Riva, MD

The compound LDK378, a highly selective inhibitor of ALK, has been granted “Breakthrough Therapy Designation” by the FDA for the treatment of patients with ALK-positive metastatic non-small cell lung cancer (NSCLC) who have already received treatment with crizotinib (Xalkori).

The designation was granted based on positive early data on LDK378. Initial results obtained from a phase I study showed a significant response rate in patients with ALK-positive NSCLC who had progressed on treatment with or had become intolerant to crizotinib, an FDA-approved treatment for this type of lung cancer.

According to a statement from Novartis Oncology, the manufacturer of the drug, a response rate of 80% was observed in the study, which included complete response, partial response, and unconfirmed partial response to the drug. Dose-limiting toxicities reported in the study included diarrhea, vomiting, nausea, and dehydration. The results were presented at the 2012 European Society of Medical Oncology Congress in Vienna, Austria.1

“LDK378 is a strong example of our research approach, which focuses on identifying the underlying cause of disease pathways,” said Alessandro Riva, MD, Global Head of Oncology Development & Medical Affairs for Novartis Oncology, in a statement. “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-positive NSCLC.”

Drugs can receive a breakthrough therapy designation as part of the 2012 FDA Safety and Innovation Act (FDASIA). According to the act, drugs like LDK378 can receive the designation if the drug is designed to treat a life-threatening condition and early evidence shows that endpoints such as survival are significantly improved over existing therapies.

The act is designed to expedite the review process by allowing for more meetings to take place during the development process, potentially reducing the number of patients required to enroll in further clinical trials, and shortening the length of those trials when possible. In the case of LDK378, Novartis Oncology can still seek fast-track designation, accelerated approval, and priority review for the drug during the review process.

According to Novartis Oncology, two phase II trials are currently enrolling patients, and several phase III trials are expected to be initiated later this year. The first regulatory filing is expected in 2014.


1. Shaw AT, Camidge R, Felip E, et al. Results of a first-in-human phase I study of the ALK inhibitor LDK378 in advanced solid tumors. Vienna, Austria: European Society for Medical Oncology; September 30, 2012. Abstract 440.



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