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FDA Breakthrough Therapy Designation: Merck Gets Accelerated Approval For First Anti-PD-1 Therapy

September 5, 2014

On September 4th, Merck announces that the FDA approves Keytruda (Pembrolizumab) for patients with unresectable/metastatic melanoma, disease progression, and have BRAF V600 mutation, following treatment with Yervoy (Ipilimumab) and a BRAF inhibitor. The indication receives accelerated approval “based on tumor response rate and durability of response”. Keytruda is given by infusion every 3 weeks.

Keytruda receives the coveted FDA Breakthrough Therapy Designation (BTD) in April 2013 for melanoma.

Keytruda is the first in a new class of cancer drugs, PD-1 blocking drugs, receiving approval in the U.S. “It is the first approved drug that blocks a cellular pathway known as PD-1, which restricts the body’s immune system from attacking melanoma cells”. Merck is executing ongoing Phase II and III clinical studies in advanced melanoma, which are designed to provide further positive evidence for Keytruda.

The FDA approves the following 5 melanoma orphan drug therapies since 2011 (Keytruda is the 6th) :

•   BMS’ Yervoy (Ipilimumab – 2011)

•   Merck’s Sylatron (Peginterferon alfa-2b – 2011)

•   Genentech’s Zelboraf (Vemurafenib – 2011)

•   GSK’s Tafinlar (Dabrafenib – 2013)

•   GSK’s Mekinist (Trametinib – 2013).

** The combination of Tafinlar + Mekinist is approved by the FDA in early 2014 for melanoma.

Per the Merck 09/04/14 news release, the company plans on making Keytruda available within 1 week. According to a New York Times article, Keytruda will cost about $12,500/month or approximately $150,000/year.



FDA BTD Approval Chart

FDA BTD Statistics Chart.

Please Note: “Icon Announcer” by Orion 8 [Public domain] | Wikimedia Commons.

Copyright © 2012-2014, Orphan Druganaut Blog. All rights reserved.

  1. The drug’s price tag may raise some flags with the government. Recently, Gilead Sciences, Inc.’s (GILD) Sovaldi came under scrutiny of various health insurers.

    • Thanks for visiting the Orphan Druganaut Blog. Yes, drug pricing for orphan drugs & even for HCV drug Sovaldi has been under criticism since it was approved. Thanks again, Ann

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