Revolutionary breakthrough! Clinical application and market price impact of RET targeted therapy platinib (updated in 2025)

Platinib capsule (Gavreto) is a new type of targeted drug, mainly used to treat metastatic RET fusion-positive non-small cell lung cancer (NSCLC) and RET fusion-positive thyroid cancer. With the rapid development in the field of cancer treatment, platinib, as a new development, has attracted widespread attention for its clinical efficacy and market price. This article will conduct an in-depth discussion of the indications, usage and dosage, mechanism of action, clinical efficacy and market price of Platinib, and analyze its importance in clinical application in light of current events.

Keywords and aliases

Pralsetinib capsules | Pralsetinib | Gavreto | RET fusion | Non-small cell lung cancer | Thyroid cancer | No tissue limit | Usage and dosage | Safety | Price

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1. Drug Positioning: Targeting Program for RET Fusion

Platinib is an oral, small molecule inhibitor that specifically targets RET gene fusions or mutations. It is indicated for adults with RET fusion-positive metastatic non-small cell lung cancer (NSCLC) and patients 12 years and older with RET fusion-positive thyroid cancer who require systemic therapy and are not responsive to RAI.

1. RET fusion-positive non-small cell lung cancer : FDA approved in 2020, and subsequently converted to routine approval.

Platinib is suitable for adult patients confirmed to have RET gene fusion. This type of lung cancer usually responds poorly to traditional chemotherapy and radiotherapy. Targeted therapy has become a new treatment option, and the use of platinib provides patients with a better chance of survival.

2. RET fusion-positive thyroid cancer : passed FDA accelerated approval in 2021.

The drug is suitable for patients 12 years of age and older with advanced or metastatic RET fusion-positive thyroid cancer. These patients often require systemic therapy and are refractory to radioactive iodine therapy. Platinib is also indicated for patients with advanced or metastatic RET-mutated medullary thyroid cancer (MTC) who require systemic therapy.

2. Recommended dosage and administration method: 400 mg/day, the rule is more efficient

1. Pre-medication testing: “necessary threshold” for RET mutation

The efficacy of platinib is highly dependent onconfirmation of RET mutation status. Using next-generation sequencing (NGS) technology for detection, its sensitivity (>95%) and specificity (>99%) are significantly better than FISH and PCR methods. For patients with thyroid cancer, RET fusion and point mutations (such as M918T, C634W) need to be detected at the same time.

2. Recommended dosage and administration method

Standard dose:400mg once a day, taken orally on an empty stomach (fasting within 2 hours before taking the medicine and 1 hour after taking the medicine).

Dose adjustment strategy:

First dose reduction:300mg once daily (for grade 3 hypertension or neutropenia);

Second dose reduction:200 mg once daily (for grade 4 liver function abnormalities or interstitial lung disease);

Discontinuation criteria: Patients who cannot tolerate 100 mg once daily need to permanently discontinue the drug.

3. Medication for special groups of people

Hepatic Impairment: No dose adjustment is required for mild hepatic impairment (Child-Pugh Class A); it is contraindicated in patients with moderate to severe hepatic impairment.

Elderly patients:Patients over 65 years old do not need to adjust the dose, but need to strengthen blood pressure and electrolyte monitoring.

This straightforward dosing strategy is easier for patients to remember and improves treatment compliance.

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3. Mechanism of actionBrief description: Highly selectiveRET inhibitoryagent

Platinib covalently bindsto the ATP binding pocket of RET kinase, inhibits the phosphorylation of wild-type RET and oncogenic mutants (such as KIF5B-RET, CCDC6-RET), and blocks the downstream MAPK and PI3K/AKT signaling pathways. Its core advantages include:

1. Super selectivity

Platinib’s IC50 value forRET is 0.4nM, which is more than 150 times lower than traditional multi-kinase inhibitors (such as cabozantinib, IC50=61.9nM), significantly reducing off-target toxicity. In cell experiments, Platinib inhibited RET at concentrations that were 14-fold, 40-fold, and 12-fold lower than VEGFR2, FGFR2, and JAK2, respectively.

2. Overcoming drug resistance

Aiming atRET V804L/M mutation (the main cause of traditional drug resistance), platinib can still maintain inhibitory activity (IC50=5nM), providing a new treatment option for drug-resistant patients.

3. Blood-brain barrier penetrability

InKIF5B-RET driven intracranial tumor mouse model, platinib significantly prolonged survival. Although the mechanism is complex, we only emphasize here: high selectivity means effective target inhibition and relatively reduced side effects.

IV. Safety analysis: common reactions are controllable, but serious risks need to be paid attention to

The most common side effects include: musculoskeletal pain, constipation, hypertension, diarrhea, fatigue, edema, fever, and cough (included in the label). Also observed in actual clinical studies:

Elevated liver enzymes (AST/ALT) are a common phenomenon

Rarely, pneumonia or interstitial lung disease occurs

Rare but serious conditions such as myolysis or drug-related pneumonia

ARROW data showed that, apart from a few adverse events, the overall safety profile was acceptable and manageable with monitoring.

5. Market price and economic analysis

1. Original drug price

Currently, the original drug Platinib has been launched in China, but it has not yet been included in the medical insurance system. Common packaging specifications are 100mg*60 pills and 100mg*120 pills. The price ranges from 20,000 to 60,000 yuan, and the specific price varies by region.

In the international market, the price of platinib is more expensive.The price of a 60-pill package fluctuates between 40,000 to more than 100,000 yuan. Such high prices place a considerable financial burden on patients.

2. Market prospects of generic drugs

At present, some companies have begun to develop generic drugs of Platinib, whose ingredients are basically the same as the original drugs on the market. For example, the 120-pellet generic drug produced by Lucius Pharmaceuticals in Laos is priced at more than 3,000 yuan. The emergence of generic drugs provides patients with more choices and helps alleviate financial pressures.

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VI. Summary of efficacy: Cancerresponses significantly and has good sustainability

1. Non-small cell lung cancer (NSCLC)

1) First-line treatment: For patients with RET fusion-positive locally advanced or metastatic NSCLC, platinib has been included in the NCCN Guidelines (2025 Edition) as a Class I recommendation. Clinical data shows that the objective response rate (ORR) of newly treated patients is 71%-78%, and the median progression-free survival (mPFS) is 12.6-14.7 months, which is significantly better than traditional platinum-based chemotherapy (ORR is about 30%, mPFS is about 5.6 months).

2) Second-line treatment: For patients who have previously failed platinum-based chemotherapy,ORR is 57%-63%, mPFS reaches 9.1-11.2 months, providing durable survival benefit for drug-resistant patients.

2. Thyroid cancer (TC)

Radioactive iodine-refractoryRET fusion-positive TC: For patients aged 12 years and above who require systemic therapy and are ineffective for radioactive iodine, platinib has an intracranial ORR of 58% and a median PFS of 18.4 months, making it a key treatment option for patients with brain metastases.

RET-mutant medullary thyroid cancer (MTC): In treatment-naive patients, the ORR reached 73%, and the median duration of response (DoR) did not reach the endpoint; for patients who had previously failed treatment with cabozantinib or vandetanib, the ORR still reached 60%, and the DoR reached 22.1 months.

In July 2025, China's National Medical Products Administration (NMPA) approved platinib for the first-line treatment of RET fusion-positive NSCLC, filling the gap in domestic RET-targeted therapy. Previously, the drug has been approved in China for the late-line treatment of RET fusion-positive NSCLC and RET-mutant MTC.

Overseas reference links:

FDA Label for GAVRETO (Pralsetinib)

NCCN Guidelines for Non-Small Cell Lung Cancer (2025 Version)

ESMO Clinical Practice Guidelines for Thyroid Cancer (2025)

ARROW Study Results (NEJM, 2024)

Global Pricing Database for Pralsetinib (Drugs.com)