FDA Grants Pfizer's Xalkori Breakthrough Therapy Status for Two New Indications

Pfizer Inc. (NYSE:PFE) announced on 5/29/18 that the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy designation for Xalkori (crizotinib) for the treatment of patients with metastatic non-small cell lung cancer (NSCLC) with MET exon 14 alterations with disease progression on or after platinum-based chemotherapy. The FDA also granted Breakthrough Therapy designation for Xalkori for the treatment of patients with relapsed or refractory systemic anaplastic large cell lymphoma (ALCL) that is anaplastic lymphoma kinase (ALK)-positive.

“Biomarker-driven therapies have changed the way we treat cancer, helping to ensure that patients receive the right medicine for their disease,” said Mace Rothenberg, M.D., chief development officer, Oncology, Pfizer Global Product Development. “These Breakthrough Therapy designations for Xalkori exemplify our commitment to precision medicine development and delivering medicines that have the potential to transform the lives of patients whose cancers carry these genomic alterations.”

Xalkori is currently approved in the U.S. for the treatment of patients with metastatic NSCLC whose tumors are ALK-positive or ROS1-positive as detected by an FDA-approved test. Xalkori became a first-line standard of care for ALK-positive metastatic NSCLC in its first approved indication and has proven to be a practice-changing treatment for patients with ALK-positive and ROS1-positive NSCLC, globally. It is the only FDA-approved treatment indicated for both ALK-positive and ROS1-positive metastatic NSCLC. If approved in patients with metastatic NSCLC with MET exon 14 alterations, Xalkori will be the only TKI with demonstrated efficacy in three separate biomarker-driven indications in NSCLC.

The FDA’s Breakthrough Therapy designation is intended to expedite the development and review of a medicine if it is intended to treat a serious or life-threatening disease and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over existing therapies. 

The recent news for Xalkori has not all been great. On May 17, 2018 Genentech relesased results from a Ph III trial comparing its Alecensa (alectinib) to  Pfizer‘s Xalkori (crizotinib) in late-stage non-small cell lung cancer (NSCLC). These results of this long term (two years) study showed Alecensa provided a significant advantage over Xalkori in disease progression and risk of death.