Is Pralsetinib worth buying? Latest medication and price reference for patients with RET gene fusion lung cancer

1. Hot topic background:RETNew breakthroughs in targeted therapy attract attention

In recent years, targeted drugs have become the core direction of cancer treatment, and RETgene fusion, as a key tumor driver mutation, has received more and more clinical attention. At the end of 2024, with the release of a number of clinical data, the RET inhibitor pralsetinib (Pralsetinib) once again became the focus of the medical community.

This drug has outstanding clinical performance worldwide and is known as another "precision targeting representative" after ALK and ROS1. Many domestic patients have been discussing on social platforms: "Can platinib be used for RET mutations?", "Is platinib effective?" - these discussions have also promoted people's attention and demand for this drug.

This article will provide a detailed analysis of Platinib’s clinical efficacy, medication guidelines, medical insurance and price comparison to help patients gain a deeper understanding of this emerging targeted drug.

2. Introduction and mechanism of action of platinib

Pralsetinib (Pralsetinib, trade name: Gavreto) is produced by Blueprint MedicinesCo-developed with Roche (Roche), it is a highly selective RET inhibitor. It inhibits the growth and spread of tumor cells by blocking the signaling pathway of RETfusion or RET point mutation.

RETFusions are most common in two types of cancer:

1.About1-2%exist in patients with non-small cell lung cancer (NSCLC)RETFusion;

2.About 10-20%of papillary thyroid carcinomas and medullary carcinomas have RET mutations.

Platinib is highly targeted and can precisely bind to theRET receptor kinase, avoiding the adverse reactions caused by traditional chemotherapy and multi-target inhibitors in the past, and providing patients with a milder and more efficient treatment option.

3. Clinical efficacy: significantly prolong survival and remission period

In the internationally renowned ARROW study, platinib demonstrated excellent efficacy in RET fusion-positive non-small cell lung cancer (NSCLC) patients:

The objective response rate (ORR) reached 72%;

The disease control rate (DCR) is as high as 90% or above;

Some patients' tumors shrank by more than80% or even achieved complete remission.

For advanced patients who have previously received chemotherapy, platinib also shows significant efficacy, with the median progression-free survival (PFS) reaching 16.5 months, which is much better than traditional treatment options.

Platinib has also made breakthrough progress in patients with thyroid cancer. ARROWTrial data show that in RETmutated medullary thyroid cancer (MTC) patients, the ORR of platinib is 60-70%, and some patients maintain long-term remission and their quality of life is significantly improved.

This series of data shows that platinib can not only quickly control tumor progression, but also bring new survival opportunities to patients in the late stage of drug resistance.

4. Medication Guidelines and Recommended Plans

Platinib is an oral targeted drug that is easy to use.

Recommended dose: 400mg once daily.

Dosage time: It is recommended to take it on an empty stomach, or at least 1 hour before a meal and 2 hours after a meal to ensure stable drug absorption.

Treatment cycle: Continuous daily administration until disease progression or intolerable toxicity occurs.

Clinically, doctors usually adjust the dose individually based on the patient'sRET mutation type, disease stage, previous medication history, and comorbidities. Liver function, blood pressure and routine blood tests need to be monitored regularly during treatment to prevent mild side effects (such as hypertension, elevated transaminases, fatigue, etc.).

5. Safety and resistance issues of platinib

Compared with early multi-targetTKI drugs, platinib has milder side effects and higher safety. Common adverse reactions include:

1.Mild to moderate hypertension;

2.Elevated liver enzymes;

3.Anemia or fatigue;

4.Some patients developed mild rash.

Long-term studies have shown that most of these adverse reactions can be recovered by reducing the dose or discontinuing the drug without affecting the continuation of the treatment course. As for the drug resistance problem, no universal drug resistance mechanism has been discovered in clinical practice, but some studies are exploring combination treatment options (such as combination with immune checkpoint inhibitors) to extend the treatment cycle of patients.

6. Domesticforeignsales and price status

As of2025In 2019, the original drug Platinib was officially launched in mainland China, but it has not yet been included in the national medical insurance directory, and patients still need to purchase it at their own expense.

Domestic price reference: Common specifications are 100mg 2More than 10,000 yuan, and in some areas even as high as 60000 yuan.

Overseas price reference: In the European and American markets, the price of platinib 60 tablets ranges from 4 to more than 100,000 yuan.

Situation of generic drugs: Lao pharmaceutical companies have launched generic drugs of Platinib. The ingredients are basically the same as the original drug. The price of 120 tablets is about 3,000 yuan, which greatly reduces the burden on patients.

For patients on long-term treatment, the price difference means significant financial pressure. Because of this, more and more patients are beginning to pay attention to legal import channels or overseas medical cooperation projects to achieve a balance between economy and efficacy.

7. Medical insurance and accessibility issues

Although platinib is currently on the market, because it has not yet been included in the medical insurance system, the threshold for use by domestic patients is still high. Relevant policies are being advanced and are expected to be included in the national medical insurance directory through negotiations in the future to reduce the financial burden on patients.

During the transition period while waiting for medical insurance coverage, patients can choose the following ways to reduce costs:

Consult through the hospital pharmacy"Specially approved drugs" or clinical research projects;

Pay attention to the overseas generic drug market, but be sure to choose legal and formal channels;

Apply for assistance programs from cancer charities or patient mutual aid foundations.

8. Real patient feedback and clinical experience

Multiple patient feedbacks from domestic and foreign countries show that platinib can quickly control symptoms after use:

Most patients are taking medicine2~4Tumor shrinkage appears within weeks;

Symptoms such as dyspnea and cough were significantly improved;

Among patients with long-term use, some maintain remission for more than two years.

In social media and patient mutual aid groups, patients generally comment on it as a cost-effective targeted drug that is “convenient to take orally, has mild side effects and has stable effects”. However, some patients also mentioned practical problems such as high prices and complicated drug purchasing channels.

9. Future prospects and research and development directions

Platinib is currently undergoing multiple expansion studies globally, including:

RETTreatment of positive pancreatic cancer and cholangiocarcinoma;

RETDevelopment of second-generation inhibitors for drug-resistant mutations;

Combination drug strategy with immunotherapy and chemotherapy.

With the accumulation of data and expansion of indications, Platinib is expected to become the core drug for targeted treatment of multiple cancer typesRET. If generic drugs can be included in medical insurance and promoted in the future, it will greatly improve patient accessibility.

The emergence of platinib marks thatRET targeted therapy has entered a new stage. It not only extends the survival time of patients with advanced cancer, but also brings new options to people who cannot tolerate traditional treatments.

However, the high price is still the biggest obstacle to the popularization of platinib in China. With the advancement of medical insurance negotiations and generic drug production, this situation is expected to improve in the future. Whether it is domestic original drugs, overseas imported versions or generic drugs, the key is to choose formal channels, safe medication, and scientific management, so that targeted therapy can truly benefit more patients.

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References:

1.ARROW Trial Clinical Data – ClinicalTrials.gov

2.FDA Drug Approval for Pralsetinib – FDA.gov

3.Blueprint Medicines Official – https://www.blueprintmedicines.com

4.NCCN Guidelines for RET+ Lung Cancer – NCCN.org

5.ESMO 2024 Annual Meeting – RET Pathway Update Report