Enacted as part of the 2012 FDA Safety and Innovation Act (FDASIA), Breakthrough Therapy designation is intended to expedite the development and review of a potential new 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 Breakthrough Therapy designation is distinct from the FDA’s other mechanisms to expedite drug development and review.
ALK gene rearrangement is a genetic alteration that drives the development of lung cancer in some patients.
Due to additional mutations that the tumor may acquire during treatment, disease progression remains a challenge in patients with ALK-positive metastatic NSCLC.
Mace Rothenberg, MD, chief development officer, Oncology, Pfizer Global Product Development, said: “This regulatory designation recognizes the potential for lorlatinib to provide an important treatment option for patients with ALK-positive NSCLC whose cancers have progressed despite treatment. Pfizer’s rapid development of lorlatinib reflects a commitment to developing biomarker-driven therapies to meet the evolving needs of patients.”
“We look forward to working with the FDA to accelerate the development of this therapy.”
The Breakthrough Therapy designation is supported by the efficacy and safety data of an ongoing Phase 1/2 clinical trial of lorlatinib, which includes patients with ALK-positive NSCLC who were previously treated with one or more ALK inhibitors.
Additionally, the Phase 3 CROWN study (NCT03052608) recently began enrolling patients. CROWN is an ongoing, open label, randomized, two-arm study comparing lorlatinib to crizotinib in the first-line treatment of patients with metastatic ALK-positive NSCLC.