FDA Grants Accelerated Approval to Pralsetinib for Expanded Indications in RET-Altered Thyroid Cancers

Recently, the U.S. Food and Drug Administration (FDA) accelerated the approval of the expanded indication for the innovative targeted RET inhibitor pralsetinib, for the treatment of patients with thyroid cancer harboring RET alterations. This isTumorAnother Important Milestone in the Field of Precision Medicine.

Pralsetinib, developed by Blueprint Medicines, is an oral, once-daily, potent and highly selective RET inhibitor. Currently, the U.S. FDA has approved pralsetinib for three indications: for the treatment of adult patients with RET fusion-positive non-small cell lung cancer (NSCLC); for patients aged 12 years and older with advanced or metastatic RET-mutant medullary thyroid cancer (MTC) requiring systemic therapy; and for patients with advanced or metastatic RET fusion-positive thyroid cancer requiring systemic therapy who are radioactive iodine-refractory. In addition, pralsetinib has beenFDAGranted “Breakthrough Therapy Designation”.

It is understood that CStone Pharmaceuticals holds the rights to the development and commercialization of pralsetinib in Greater China. Currently, China’s National Medical Products Administration (NMPA) accepted the marketing application for pralsetinib as a Class 1.1 innovative drug on September 4 this year and included it in the priority review program for the treatment of patients with RET fusion-positive non-small cell lung cancer (NSCLC) who have previously received platinum-based chemotherapy. Notably, on September 30 this year, pralsetinib was successfully introduced into the Boao Lecheng International Medical Tourism Pilot Zone in the Hainan Free Trade Port. This makes it the only market available outside the United States where the drug has been launched, allowing domestic patients to apply for early access in Boao Lecheng.

In addition, CStone Pharmaceuticals has conducted registrational clinical studies in China on pralsetinib as first-line treatment for RET fusion-positive non-small cell lung cancer (NSCLC) and medullary thyroid carcinoma. Meanwhile, the efficacy of pralsetinib in other advanced solid tumors harboring RET alterations is being further explored. To date, pralsetinib has demonstrated broad and durable anti-TumorPotent activity with boundless potential. We look forward to the early approval and market launch of pralsetinib, an innovative RET inhibitor, in China, so that it can benefit more patients as soon as possible.

In China, the incidence of thyroid cancer has been rising year by year. Relevant reports indicate that the incidence rate is approximately 14.6 per 100,000 people, with medullary thyroid carcinoma (MTC) accounting for about 2%-4% of all thyroid cancer cases. MTC is highly malignant, prone to distant metastasis, and associated with poor therapeutic outcomes in advanced-stage patients. Approximately 60% of MTC cases harbor RET gene mutations, with this proportion reaching as high as 90% in advanced-stage disease. RET gene mutations can be regarded as the “root cause” of medullary thyroid carcinoma. For advanced MTC with a 90% prevalence of RET mutations, precise inhibition of RET mutations is crucial and will further improveTumorRemission rate, improving patient prognosis.

The “birth” of the innovative targeted drug pralsetinib has broken the situation of multi-target “shotgun” approaches in medullary thyroid carcinoma (MTC). It can highly selectively and precisely target RET mutant genes, offering broader and more durable anti-Tumoreffect, and by inhibiting primary and secondary mutations, pralsetinib is expected to overcome and prevent the development of clinical resistance.

Beyond the field of thyroid cancer, pralsetinib will also bring new hope to patients with RET fusion-positive non-small cell lung cancer (NSCLC). Currently, targeted therapies against widely prevalent driver gene mutations such as EGFR, ALK, and ROS1 have already been approved and marketed. RET fusion is a recently identified driver gene in lung cancer; patients with RET fusion-positive NSCLC account for approximately 1–2% of cases and are more commonly found among non-smokers. Based on the large patient population of lung cancer, it is estimated that there are approximately 11,000 newly diagnosed RET-positive lung cancer patients annually in China. For patients with RET mutations, the likelihood of harboring other oncogenic driver genes is low,DiagnosisAt that time, most patients were already in the advanced stages of the disease, posing significant challenges to diagnosis and treatment.

Currently, the standard treatment for RET fusion-positive non-small cell lung cancer remains predominantly chemotherapy, which yields suboptimal efficacy, leaving a substantial unmet clinical need. Pralsetinib, as a therapy specifically designed for RET gene alterationsTumorA highly selective RET inhibitor developed for patients will provide new hope for patients with RET fusion-positive non-small cell lung cancer.

As a biopharmaceutical company focused on the development and commercialization of innovative immuno-oncology and precision medicine therapies, CStone Pharmaceuticals has assembled a world-class management team with extensive experience in new drug development, clinical research, and commercialization. Currently, CStone Pharmaceuticals has established a pipeline of 16TumorA robust pipeline of candidate drug products, with five late-stage candidates currently in pivotalClinical Trialor registration stage. With an experienced management team, a robust pipeline, a business model focused on clinical development, and ample funding, CStone Pharmaceuticals aims to become a globally renowned biopharmaceutical company, leading the way in the fight against cancer.