The US FDA's breakthrough therapy program is helping tissue-agnostic cancer therapy sponsors design clinical trials and navigate a regulatory apparatus designed for the dominant tissue histology-based paradigm for oncology.
Oncology: Tissue-Agnostic Indications Advance Under US FDA's Breakthrough Umbrella
Merck's Keytruda may not be only oncologic with indication for cancer patients defined by molecular signature, not tissue of origin, for long: Ignyta's entrectinib and Loxo Oncology's larotrectinib are positioned for breakthrough-designated tissue-agnostic NDA submissions in NTRK fusion-positive cancers in 2018.