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Merck Receives FDA Breakthrough Therapy Designation

April 24, 2013

Merck announces April 24, 2013, that investigational antibody therapy drug Lambrolizumab (MK-3475) receives FDA Breakthrough Therapy designation for the treatment of patients with advanced melanoma.

Lambrolizumab targets PD-1, an immune checkpoint receptor, that “helps to confer immune resistance for some cancers allowing tumor cells to grow and proliferate unchecked .. is a monoclonal antibody designed to target PD-1 to disrupt the role PD-1 plays in resisting the immune system”.

In November 2012, at the 9th International Congress of the Society for Melanoma Research (SMR), early-stage interim data from a single-arm, open-label Phase Ib study of Lambrolizumab are presented.  The drug is given to 85 patients with advanced inoperable metastatic melanoma. The following are the early-stage interim study results presented at the meeting :

•    Approximately 51% of patients with advanced melanoma experience tumour shrinkage after treatment
•    Approximately 10% have no detectable cancer after treatment as assessed by imaging techniques.

Lambrolizumab is being investigated in a global, randomized, Phase II clinical trial to evaluate the drug versus standard chemotherapy for participants with advanced melanoma, whose disease has progressed after prior therapy. The drug is also being studied in other cancer types, including Non-Small Cell Lung Cancer (NSCLC).  According to, analysts at Leerink Swann estimate that if the drug is approved, “Lambrolizumab could generate peak sales of $450 million in the melanoma indication by 2023”.

Advanced melanoma accounts for :

•    More than 80% of skin cancer-related deaths
•    1%-2% of all cancer deaths in the US
•    Approximately 9,180 deaths in 2012 in US.

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.

Please Note #1: The Orphan Druganaut Blog has decided to include a Post on drug Lambrolizumab even though it isn’t an orphan drug since the Blog has been following the new FDA Breakthrough Therapy designation from 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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