NorthWest Biotherapeutics Inc (NWBO)

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Keytruda (pembrolizumab) was first approved by the FDA on September 4, 2014, for advanced melanoma, under accelerated approval based primarily on data from a single large Phase 1b trial known as KEYNOTE-001 (NCT01295827). This trial, which started in 2011, was a multi-cohort, adaptive study enrolling over 1,200 patients with advanced solid tumors, including melanoma and non-small cell lung cancer (NSCLC).

It included multiple expansions and cohorts testing different doses and indications.

Prior to approval, clinical development was unusually rapid and relied heavily on this one pivotal trial rather than the traditional sequence of separate Phase 1, 2, and 3 studies. KEYNOTE-001 served as the key source of safety and efficacy data (tumor response rate and durability), with subsets of patients (e.g., 173 ipilimumab-refractory melanoma patients) supporting the initial approval.

This streamlined approach was enabled by breakthrough therapy designation and the drug's strong early signals in a high-unmet-need area. In short, essentially one major clinical trial (KEYNOTE-001 with its cohorts) supported the initial approval,