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Novartis Receives FDA Breakthrough Therapy Designation

March 15, 2013

Novartis announces March 15, 2013, that their compound LDK378 receives FDA Breakthrough Therapy designation for treatment of  patients with Anaplastic Lymphoma Kinase positive (ALK+) metastatic  Non-Small Cell Lung Cancer (NSCLC), who have progressed during treatment with, or were intolerant to Pfizer’s orphan drug Xalkori (Crizotinib). The FDA designation is based on positive early data in patients with ALK+ NSCLC who have previously been treated with Crizotinib. Patients with ALK+ NSCLC have few treatment options. This is the 5th Breakthrough Therapy designation given by the FDA in 2013 (the 3rd designation for oncology) :

  1. Two are awarded to Vertex Pharmaceuticals for expanding the uses of its Cystic Fibrosis drug Kalydeco
  2. Two are awarded to Pharmacyclics and Janssen Research & Development for the cancer therapy drug Ibrutinib.

Alessandro Riva, Global Head, Oncology Development & Medical Affairs at Novartis Onclogy, says that, “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.”

As discussed in previous Orphan Druganaut Blog Posts, the new FDA Breakthrough Therapy Designation is for the purpose of speeding up the development and review of new drugs. In order for a drug to be designated as a Breakthrough Therapy, the sponsor must provide, per Section 902 of FDASIA :

  1. Evidence that the drug is intended , alone or in combination with 1 or more other drugs, to treat a serious or life-threatening disease or condition, and
  2. Preliminary clinical evidence indicating that the drug may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints, such as substantial treatment effects observed early in clinical development.

Results from a Phase I study of LDK378 in 88 patients shows a “response rate (including Complete Response [CR], Partial Response [PR] and unconfirmed PR) of 80% was observed in the patients who had experienced disease progression after Crizotinib treatment.” Novartis has initiated two Phase II clinical trials and plans to initiate several Phase III trials later in 2013. First regulatory filing is anticipated by early 2014.


  1. Is 85 – 90% of all cases of lung cancer
  2. 3 – 8% of NSCLC patients have the ALK gene mutation
  3. Limited # of treatment options for ALK+ NSCLC
  4. Tend to be non-smokers and younger than NSCLC patients without the ALK gene mutation.

Including the biomarker test with an orphan drug is very useful for maintaining a drug’s share of the market in the face of competition. Pfizer’s Xalkori is developed in parallel with a companion diagnostic to identify the presence of the ALK fusion gene in a subset of non-small cell lung cancer (NSCLC) patients. Pfizer partnered with Abbott Molecular to develop the test. The Vysis ALK Break Apart FISH Test receives FDA approval at the same time that Xalkori receives FDA approval in August 2011.

References for LDK378

Novartis Pipeline

Please Note #1: The Orphan Druganaut Blog has decided to include a Post on drug LDK378 even though it isn’t an orphan drug, because the Blog has been following the new FDA Breakthrough Therapy designation since the beginning of 2013.

Please Note #2: FDA Official Logo from FDA website.

Copyright © 2012-2013, Orphan Druganaut Blog. All rights reserved.

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