Keytruda's First FDA Approval
Keytruda (pembrolizumab) received its initial FDA accelerated approval on September 4, 2014, for unresectable or metastatic melanoma in patients whose tumors express PD-L1 or who have progressed after ipilimumab and, if BRAF V600 mutation positive, a BRAF inhibitor.[1]
What Was the Trial Behind It?
Approval stemmed from the KEYNOTE-001 phase 1 trial, which showed 33% objective response rate in 89 PD-L1-positive melanoma patients, with 8 complete responses.[1]
How Did Indications Expand from There?
FDA granted subsequent approvals for melanoma subtypes (e.g., ipilimumab-refractory in December 2014), then non-small cell lung cancer (October 2015), classical Hodgkin lymphoma (May 2017), and over 30 others by 2024, including head/neck, urothelial, and MSI-H cancers.[1][2]
Why Accelerated Approval Initially?
The agency used accelerated approval based on tumor response rates and durability as surrogate endpoints, later confirmed by overall survival data in follow-on studies.[1]
Current Status of Original Indication
The melanoma approval converted to full approval in 2016 after KEYNOTE-002 and KEYNOTE-006 trials confirmed progression-free and overall survival benefits.[1]
[1]: FDA Approval History for Keytruda
[2]: Drugs@FDA - Pembrolizumab