Pfizer receives FDA approval for XALKORI capsules to treat ALK-positive NSCLC

Pfizer Inc. announced today that the U.S. Food and Drug Administration (FDA) has approved XALKORI® (crizotinib) capsules, the first-ever therapy targeting anaplastic lymphoma kinase (ALK), for the treatment of patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) that is ALK-positive as detected by an FDA-approved test. The effectiveness of XALKORI is based on objective response rates (ORR) and, as XALKORI received accelerated approval from the FDA, Pfizer is conducting post-marketing clinical trials to further evaluate its clinical benefit.

"Overall, lung cancer is responsible for more deaths each year worldwide than any other type of cancer. XALKORI is an advance in the treatment of this devastating illness, providing a new therapeutic option for a subset of patients with the disease," said Ian Read, president and chief executive officer of Pfizer. "The acceleration, collaboration and critical focus of the XALKORI clinical development program reflect Pfizer's Precision Medicine approach to advancing our pipeline and strengthening our innovative core to deliver medicines that matter most."

Aligned with the FDA's latest guidance on targeted therapies and companion diagnostics, the Company worked closely with the FDA and partnered with Abbott Molecular's business in Pfizer's clinical studies to ensure the simultaneous review and approval of XALKORI along with a diagnostic test, Abbott Molecular's Vysis ALK Break Apart FISH Probe Kit, to identify presence of the ALK fusion gene. The simultaneous approval of XALKORI in parallel with Abbott Molecular's ALK FISH Test marks the first time a Pfizer oncology drug or any lung cancer medication was developed and approved in parallel with a diagnostic test.

XALKORI is available immediately through a number of specialty pharmacies. Patients prescribed XALKORI can call 1-877-744-5675 for assistance accessing the medication. For more information about the FDA-approved ALK test, call (855) TEST-ALK (837-8255).

"By truly understanding the underlying genetic drivers of NSCLC, such as ALK, we can select patients who are more likely to respond to treatment. XALKORI provides a model for how to approach future drug development and cancer care," said Dr. Paul Bunn, professor of medicine and the James Dudley chair in cancer research at the University of Colorado, Denver. "XALKORI, the first new drug approved for lung cancer by the FDA in more than six years, represents a paradigm shift in NSCLC treatment, where we're moving away from a one-size-fits-all approach to biomarker-based treatment decisions."

In the clinical trials for XALKORI, the study design required patients' tumors to prospectively test positive for the ALK fusion gene biomarker, increasing the likelihood of response to the treatment. This method, a first for a lung cancer therapy not yet on the market, allowed researchers to observe a strong efficacy signal in a selected patient population. Preliminary epidemiology suggests that approximately 3-5 percent of NSCLC tumors are ALK-positive, translating to approximately 6,500 to 11,000 NSCLC patients in the U.S. each year.

"XALKORI represents a new chapter in personalized therapy for lung cancer, enabling physicians to provide the right treatment for the right patient," said Dr. Mace Rothenberg, senior vice president of clinical development and medical affairs for Pfizer's Oncology Business Unit. "The development of XALKORI - from publication of the discovery of the ALK fusion gene in NSCLC to FDA approval in just four years - is a remarkable feat in the oncology world and reinforces the importance of collaboration among academic research, pharmaceutical, diagnostic and regulatory organizations."

Using a targeted approach in the XALKORI registration trials, ORR of 50 and 61 percent were observed in patients with advanced ALK-positive NSCLC.

"Today's approval of XALKORI underscores the important role of molecular biomarkers in cancer treatment," said Dr. Joan Schiller, president of National Lung Cancer Partnership and chief of Hematology/Oncology, University of Texas Southwestern Medical Center. "We strongly encourage lung cancer patients to talk to their oncologists about molecular tumor testing. By having a full understanding of the molecular biology of their tumor, patients and physicians can make well-informed treatment decisions."