What does Pralsetinib do?

The U.S. Food and Drug Administration (FDA) approved pralsetinib for non-small cell lung cancer based on ARROW, a multicenter, non-randomized, open-label, multi-cohort Phase 1/2 trial. This study enrolled in separate cohorts patients with metastatic RET fusion-positive NSCLC who progressed on platinum-based chemotherapy, as well as patients with metastatic NSCLC who were treatment-naïve. Patients received 400 mg of platinib orally once daily until disease progression or unacceptable toxicity occurred.

The primary efficacy outcome measures were overall response rate (ORR) and duration of response (DOR). The efficacy was evaluated in 87 patients with RET fusion-positive non-small cell lung cancer from the ARROW cohort who had measurable disease and had previously received platinum-based chemotherapy. Platinib demonstrated an overall response rate (ORR) of 57% and a complete response (CR) rate of 5.7% in 87 patients with NSCLC who had previously received platinum-based chemotherapy, and the median duration of response (DoR) was not reached. Among 27 treatment-naïve NSCLC patients, the ORR was 70% and the CR rate was 11%.

The FDA's subsequent accelerated approval of platinib for the treatment of RET-mutated medullary thyroid cancer was based on the Phase I/II ARROW study, an open-label study designed to evaluate the safety, tolerability, and efficacy of platinib administered orally in patients with rearrangement during transfection (RET) fusion-positive non-small cell lung cancer (NSCLC), RET-mutant medullary thyroid carcinoma (MTC), RET fusion-positive thyroid cancer, and other RET-altered solid tumors.

The overall response rate (ORR) of platinib treatment was 60% in 55 patients with RET-mutated metastatic MTC who had previously received cabozantinib and/or vandetanib and had not reached the median duration of response (DoR). Among 29 patients with RET-mutated advanced MTC, cabozantinib and/or vandetanib treated naïve patients, the ORR was 66%, and the median DoR was not reached. In nine patients with RET fusion-positive metastatic thyroid cancer, platinib demonstrated an ORR of 89% and the median DoR was not reached.

It is understood that the original drug Platinib has been launched in China, but it has not yet entered the scope of medical insurance. The price of each box of 100mg*120 tablets may be around 60,000 yuan, which is expensive. The price of the European version of 100mg*60 capsules sold overseas may be around 40,000 yuan per box, and the price of the American version may be around 150,000 yuan per box (prices may fluctuate due to exchange rates). The ingredients of domestic and foreign original drugs are basically the same. There is currently no generic version of Platinib available on the market. Please consult your medical consultant for specific prices and drug details.