CStone Pharmaceuticals, a leading biopharmaceutical company focused on developing and commercializing innovative immuno-oncology therapies and precision medicines, today announces that the National Medical Products Administration (NMPA) of China has accepted the New Drug Application (NDA) of selective inhibitor GAVRETO® (Pralsetinib) with priority review designation for the treatment of the patients with advanced or metastatic RET-altered thyroid cancer. This may expand the labeled indications for GAVRETO® in China to include advanced or metastatic RET-mutant medullary thyroid cancer (MTC) who require systemic therapy, or with advanced or metastatic RET fusion-positive thyroid cancer who require systemic therapy and radioactive iodine-refractory (if radioactive iodine is appropriate).
In March 2021, the NMPA granted approval of pralsetinib under the brand name GAVRETO® for the treatment of adult patients with locally advanced or metastatic RET fusion-positive non-small cell lung cancer (NSCLC) after platinum-based chemotherapy. Discovered by CStone's partner Blueprint Medicines, GAVRETO® is the first approved selective RET inhibitor in China.
Dr. Jason Yang, Chief Medical Officer of CStone, said: "We are excited to see that this NDA has been accepted by the NMPA, which is six months in advance of our schedule. There has been a lack of precision medicines for RET-altered thyroid cancer in China and patients can only be treated with multi-targeted drugs. Study results showed that GAVRETO® had robust and durable efficacy and a well-tolerated safety profile among patients with RET-mutant MTC and RET fusion-positive thyroid cancer. We thank the NMPA for granting priority review for this indication and look forward to getting approval and hope benefit more patients in China in the future."
This NDA acceptance is based on efficacy and safety results from the global Phase 1/2 ARROW trial, designed to evaluate pralsetinib in patients with RET fusion-positive NSCLC, RET-mutant MTC, and other advanced solid tumors with RET fusions.
Results from the ARROW trial in patients with RET-mutant MTC were presented at the European Society for Medical Oncology Virtual Congress in September 2020. As of a data cutoff date of February 13, 2020, the results showed that pralsetinib had robust and durable anti-tumor activity in response-evaluable patients who received a starting dose of 400 mg once daily. In 53 patients previously treated with cabozantinib or vandetanib, the overall response rate (ORR) was 60 percent (95% CI: 46%, 74%) with one response pending confirmation, and the median duration of response (DOR) was not reached (95% CI: not reached, not reached). In 19 systemic treatment-naïve patients who were ineligible for standard therapy per the study protocol, the confirmed ORR was 74 percent (95% CI: 49%, 91%), and the median DOR was not reached (95% CI: 7 months, not reached). In 438 ARROW trial patients across RET-altered tumor types, the most common treatment-related adverse events reported by investigators (≥ 15 percent) were increased aspartate aminotransferase, anemia, increased alanine aminotransferase, hypertension, constipation, decreased white blood cell count, neutropenia, decreased neutrophil count and hyperphosphatemia.
CStone has an exclusive collaboration and license agreement with Blueprint Medicines for the development and commercialization of GAVRETO® in Greater China, which encompasses Mainland China, Hong Kong, S.A.R., China, Macau, S.A.R., China, and Taiwan, China.
About GAVRETO (pralsetinib)
GAVRETO (pralsetinib) is a once-daily oral targeted therapy approved by the NMPA of China for the treatment of adults with locally advanced or metastatic rearranged during transfection (RET) fusion-positive NSCLC after platinum-based chemotherapy.
GAVRETO has been approved by the U.S. Food and Drug Administration (FDA) for the treatment of adult patients with metastatic RET fusion-positive NSCLC as detected by an FDA approved test, adult and pediatric patients 12 years of age and older with advanced or metastatic RET-mutant MTC, and adult and pediatric patients 12 years of age and older with advanced or metastatic RET fusion-positive thyroid cancer who require systemic therapy and who are radioactive iodine-refractory (if radioactive iodine is appropriate).
GAVRETO is not approved for the treatment of any other indication in China b...
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