FDA accelerates approval of innovative RET inhibitor extension adaptation for the treatment of specific thyroid cancer patients

Blueprint Medicines and Genentech have jointly announced that the FDA has accelerated the approval of Gawreto (pralsetinib) extended adaptation for the treatment of patients with thyroid cancer with RET variants, including patients over 12 years of age with advanced or metastatic RET mutated thyroid myeloma (MTC), or patients who require systemic therapy and radioactive iodine incurable advanced or metastatic RET fusion thyroid cancer.
Gavreto is a daily oral precision therapy developed by Blueprint Medicines, designed to forcefully and selectively target RET variants that drive multiple tumor types.
Cornerstone Pharmaceuticals has been awarded a development interest in Pralsetinib in Greater China.
retactive fusion and mutation are key disease drivers for many cancer types, including non-small cell lung cancer and multiple types of thyroid cancer.
about 1-2% of patients with non-small cell lung cancer and about 10-20% of patients with thyroid papyroid cancer carry RET fusion, while about 90% of patients with advanced MTC carry RET mutations.
, cancer-causing RET fusion also occurs at lower frequencies in colorectal, breast, pancreatic and other cancer types.
Gawreto aims to selectively and powerfully target cancer-causing RET mutations, including predicting secondary RET mutations that drive tumor resistance.
has been approved by the FDA to treat metastasis RET fusion-positive non-small cell lung cancer in adult patients.
approval is based on the efficacy and safety results of a Phase 1/2 clinical trial called ARROW.
in 55 treated MTC patients with RET mutations, Gavreto achieved a total remission rate (ORR) of 60%.
ORR was 66% of the 29 primary MTC patients with RET mutations.
, the ORR was 89% in 9 RET fusion-positive thyroid cancer patients.
duration (DoR) of these patient groups has not yet been reached.
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