The Food and Drug Administration has approved the second drug that targets a certain genetic abnormality found in small percentages of patients with various solid tumors, but that represents a highly potent drug target.
Cambridge, Massachusetts-based Blueprint Medicines said Friday evening that the FDA had given accelerated approval to Gavreto (pralsetinib), a RET inhibitor, for adults with RET fusion-positive non-small cell lung cancer with or without prior therapy. The company will work with Swiss drugmaker Roche and its U.S. subsidiary, Genentech, to co-commercialize the drug in the U.S., under a previously announced deal.
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Shares of Blueprint fell 3.7% on the Nasdaq when markets opened Tuesday and remained down 3.5% early in the afternoon.
“Targeted therapies have dramatically improved care for patients with non-small cell lung cancer driven by oncogenes, including EGFR and ALK, and the approval of the selective RET inhibitor pralsetinib, or Gavreto, marks another milestone in a paradigm shift toward precision medicine,” said Dr. Vivek Subbiah, an investigator in the Phase I/II ARROW trial that led to Gavreto’s approval and medical director of The University of Texas MD Anderson Cancer Center’s Clinical Center for Targeted Therapy, in a statement.
Gavreto is the second RET inhibitor to win FDA accelerated approval, after the agency approved Eli Lilly’s Retevmo (selpercatinib) in May. However, while Retevmo won approval in RET fusion-positive NSCLC and thyroid cancer and RET mutation-positive medullary thyroid cancer, Blueprint applied for Gavreto’s approval in the latter two indications separately. The company said Tuesday that the FDA had accepted the application for the two thyroid cancer indications along with approving Gavreto in NSCLC.
At the same time, Gavreto is distinguished from Retevmo in being administered once a day, while the Lilly drug is taken twice daily. However, some analysts have previously stated that they regard the two drugs as essentially interchangeable.
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Data from ARROW showed that in 87 patients who were previously treated with platinum-based chemotherapy, there was a 57% response rate, including a 5.7% complete response rate, while median duration of response could not be estimated. In 27 patients who were treatment-naive and ineligible to receive platinum chemotherapy, the overall response rate was 70%, with an 11% complete response rate and a median nine-month duration of response. The drug is in a Phase III trial of RET fusion-positive metastatic NSCLC.
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