CStone Pharmaceuticals (Hong Kong Stock Exchange Code: 2616), a leading biopharmaceutical company focused on the development and commercialization of innovative tumor immunotherapy and precision treatment drugs, today announced that the selective RET inhibitor Pugeta® (Platin) The application for extended indications of Ni capsules has been accepted by the National Medical Products Administration (NMPA) of China and has been included in priority review.
The extended indications include late or metastatic transfection rearrangement (RET) gene mutations that require systemic treatment Medullary thyroid carcinoma (MTC), and advanced or metastatic RET fusion-positive thyroid cancer (TC) that requires systemic treatment and is refractory to radioactive iodine (if radioactive iodine is appropriate).
In March of this year, NMPA approved pratinib to be marketed under the trade name Pugeta® for the treatment of locally advanced or metastatic non-small cell lung cancer patients who have previously received platinum-containing chemotherapy for the treatment of RET gene fusion-positive adult patients.
Phuket Hua ® is China's first approved selective RET inhibitor, developed by Blueprint Medicines, a partner of CStone Pharmaceuticals.
Yang Jianxin, Chief Medical Officer of CStone Pharmaceuticals, said: “We are very pleased to see that Pugeta® has been accepted by the NMPA for its indications for thyroid cancer, which is half a year earlier than the original plan.
In China, there is no precise target for RET-mutated thyroid cancer.
To drugs, patients can only receive treatment with multi-target non-selective drugs, which have limited efficacy and poor safety.
Research data shows that in patients with RET-mutated medullary thyroid cancer and RET-fusion-positive thyroid cancer patients, Pugeta® has the performance We are grateful to NMPA for including this indication application for priority review, and look forward to the early approval of the new indication for Pugeta Hua®, which will benefit more Chinese patients.
The acceptance of the extended indications of Pugeta® is based on a global phase I/II ARROW clinical study, which aims to evaluate the presence of pratinib in RET fusion-positive non-small cell lung cancer, thyroid cancer, and other RET variants.
Safety, tolerability and efficacy in patients with advanced solid tumors.
In September 2020, at the Virtual Conference of the European Society of Medical Oncology (ESMO), the ARROW study of RET-mutant MTC patients was announced.
The results as of the data cut-off date on February 13, 2020 show that pratinib has potent and long-lasting anti-tumor activity in patients who receive an evaluable initial dose of 400 mg once daily.
Among 53 patients who had previously received cabozantinib or vandetanib, the objective response rate (ORR) was 60% (95% CI: 46%, 74%), one case of remission was to be confirmed, and the median duration of remission (DOR) has not been reached (95% CI: not assessable).
In 19 patients without systemic treatment, the confirmed ORR was 74% (95%CI: 49%, 91%), and the median DOR had not been reached (95%CI: 7 months, not evaluable).
Among the 438 tumor patients with RET mutations in the ARROW study, the most common treatment-related adverse events (≥15%) reported by the researchers were elevated aspartate aminotransferase, anemia, elevated alanine aminotransferase, hypertension, Constipation, decreased white blood cell count, neutropenia, decreased neutrophil count, and hyperphosphatemia.
Pugeta® is a potent and selective RET inhibitor developed by Blueprint Medicines, a partner of CStone Pharmaceuticals.
CStone Pharmaceuticals and Blueprint Medicines have reached an exclusive cooperation and licensing agreement to obtain the exclusive development and commercialization rights of Phuket Hua ® in the Greater China region, including Mainland China, Hong Kong, Macau and Taiwan.
Thyroid cancer is the most common endocrine malignant tumor, and its incidence has increased significantly in recent years.
According to the data released by the National Cancer Center in 2019, the incidence of thyroid cancer ranks 4th among all malignant tumors among women in urban areas in my country, and the 7th overall incidence of cancer types.
There are about 90,000 new cases of thyroid cancer in China each year, and about 6,800 deaths.
Thyroid cancer is clinically divided into papillary carcinoma, follicular carcinoma, undifferentiated carcinoma and medullary carcinoma and other pathological types.
Different types of thyroid cancer are different according to their tumor characteristics, treatment methods and prognosis.
RET fusion and activating mutations are key disease drivers in many cancer types, including NSCLC and many types of thyroid cancer.
About 10-20% of patients with papillary thyroid cancer (the most common thyroid cancer) carry RET fusion, and about 50-90% of patients with advanced MTC (about 2-5% of thyroid cancer) carry RET mutations.
There is currently no effective approved standard treatment for patients with RET mutant MTC in China.
About Pugeta® (Platinib Capsules)
Pratinib is an oral, once-a-day, potent and highly selective RET inhibitor.
It has been approved by the China National Medical Products Administration for the treatment of transfection rearrangement (RET) genes that have previously received platinum-containing chemotherapy Adult patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) with positive fusion.
The US Food and Drug Administration approved it to be marketed under the trade name GAVRETO™, which are used to treat adult patients with metastatic RET fusion-positive NSCLC confirmed by FDA-approved testing methods, and advanced or metastatic RET that requires systemic treatment Adults with mutant medullary thyroid cancer and children 12 years and older, as well as adults with advanced or metastatic RET fusion-positive thyroid cancer and children 12 years and older who require systemic treatment and are refractory to radioiodine (if applicable).
Pratinib has not been approved for other indications in China or the United States, or medical regulatory agencies in other regions have not made an approval decision for any indication of pratinib.
Pratinib is designed to selectively and effectively target oncogenic RET mutations, including secondary RET mutations that may lead to treatment resistance.
In preclinical studies, pratinib inhibits RET at a lower concentration than other drug-related kinases, including VEGFR2, FGFR2 and JAK2.
Globally, the clinical development of pratinib for the treatment of RET-fused NSCLC, various types of thyroid cancer and other solid tumor patients is underway.
The European Medicines Agency has accepted a marketing authorization application for pratinib for the treatment of RET fusion-positive NSCLC.
The FDA granted pratinib a breakthrough therapy designation for the treatment of RET fusion-positive NSCLC that has progressed after platinum-based chemotherapy, and RET mutation-positive medullary thyroid cancer that requires systemic treatment and has no alternative therapy.
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