How effective is Gilteritinib in the treatment of acute myeloid leukemia? Full analysis of price, efficacy, and medication methods

1. FLT3MutationAMLThe treatment ushered in a new situation

In recent years, the treatment field of acute myeloid leukemia (AML) has undergone earth-shaking changes. Especially driven by the continuous development of molecular classification and targeted therapy, FLT3 mutations have become an important breakthrough for precision treatment. Gilitinib (Gilteritinib), as a highly selective FLT3 inhibitor, has been proven to be effective globally. span>FLT3mutated relapsed/refractoryAML patients have significant efficacy.

In the years 2023～2025, international authoritative academic conferences (such as ASH, A SCO) and multiple real-world studies have successively announced long-term survival data and new explorations of combination therapy with geritinib, providing new treatment hope for patients with relapsed/refractory AML. As the drug is launched in China, more and more patients and doctors are paying attention to its efficacy, price and accessibility.

2. Drug mechanism: precise inhibitionFLT3mutation pathway

Gilteritinib (Gilteritinib) is a second-generation oral FLT3 inhibitor that can selectively inhibit the activity of FLT3-ITD and FLT3-TKD mutations. FLT3 is one of the most common gene mutations in AML, accounting for about 30% of them. Mutation can lead to abnormal proliferation and resistance to apoptosis of leukemia cells, becoming an important driving force for disease progression.

Compared with first-generation FLT3 inhibitors (such as Midostaurin), giritinibFLT3 has stronger inhibition, higher selectivity, and can penetrate the central nervous system. Some studies have shown that it also has certain activity in patients with central nervous system relapse. This makes it one of the important choices in the second-line or even first-line treatment of FLT3 mutated AML.

3. Global and Chinese approval progress

Giritinib received accelerated approval from the USFDA in2018 for the treatment of adult patients with relapsed or refractory AML carrying FLT3 mutations. Its approval was based on results from the pivotal ADMIRAL study, which demonstrated that gilitinib was superior to traditional chemotherapy in terms of overall survival (OS) and response rates.

Giritinib has also been approved in many regions including Japan and the European Union, becoming part of the standard treatment for FLT3mutatedAML. In recent years, multiple combination treatment options (such as combination with azacitidine, venetoclax and other drugs) have also entered the clinical trial stage, and preliminary results show that the efficacy can be further improved.

In China, giritinib has completed marketing approval, but it has not yet been included in the medical insurance catalog. Some hospitals can obtain the drug through special in-hospital procurement channels.

4. Key clinical data:ADMIRALbased on research

The approval of giritinib was based on a landmark III clinical study - the ADMIRAL study. The study included 371 patients with FLT3 mutated relapsed or refractory AML and compared the efficacy and safety of giritinib alone with standard salvage chemotherapy.

The results show:

- The median overall survival in the geritinib group was 9.3months, significantly longer than the chemotherapy group's 5.6 months;

- The complete response rate (CR/CRh) was 34% in the geritinib group and only 15% in the chemotherapy group;

- Adverse events related to giritinib treatment are relatively controllable, mainly including increased transaminases, increased creatine kinase, and mild to moderate gastrointestinal reactions.

Long-term follow-up data show that some patients achieved deep remission after treatment with giritinib, and even became a bridging treatment for subsequent hematopoietic stem cell transplantation, further improving the prognosis.

5. Indications, usage and dosage

【Indications】

Giritinib is suitable for adult patients with relapsed or refractory acute myeloid leukemia (AML) carrying FLT3 mutations. Some studies are exploring its application prospects in first-line treatment, maintenance treatment and combination treatment with other targeted chemotherapy drugs.

【Usage and Dosage】

The recommended dose is 120 mg orally daily until disease progression or intolerable toxicity occurs. During the medication period, blood routine, liver and kidney function, and creatine kinase levels should be monitored regularly, and the dose should be adjusted or treatment suspended if necessary.

In clinical use in China, the administration method of giritinib is consistent with that of the international market, but access to the drug is relatively limited, and most patients rely on the hematology department of large tertiary hospitals or formal overseas channels to purchase drugs.

6. Price information and drug purchasing channels

Currently, giritinib is available in China but has not yet been included in the medical insurance system. Due to limited procurement channels, some hospitals may not be able to purchase drugs directly, and patients need to purchase them through regular overseas pharmacies or pharmaceutical company-authorized platforms.

In foreign markets, giritinib is divided into two categories: original drugs and generic drugs:

- Original drugs: There are mainly European versions and Hong Kong versions. The price of the Hong Kong version is as high as about 100,000 yuan, which is expensive;

- Generic drugs: The price of the Laos version of giritinib ranges from 1000～2000. The ingredients are basically the same as the original drug, which reduces the huge financial burden for patients.

When patients consider purchasing drugs overseas, they should use regular licensed pharmacies or qualified third-party platforms to ensure that the sources of drugs are legal and of reliable quality, and avoid purchasing counterfeit drugs from unknown sources.

7. Safety and adverse reactions

Giritinib is generally well tolerated, and common adverse reactions include abnormal liver function, elevated creatine kinase, diarrhea, fatigue, and joint and muscle pain. Individual patients may develop differentiation syndrome (Differentiation Syndrome), which requires vigilance and timely use of glucocorticoid intervention.

In practical applications, it is recommended that patients strengthen monitoring in the early stages of treatment, especially blood routine, liver and kidney function and electrocardiogram, and dose adjustments are made if necessary. Physicians should follow guidelines to balance efficacy and safety when managing adverse reactions.

8. Clinical Application Suggestions

For patients with relapsed/refractoryAML carrying FLT3 mutations, giritinib is currently one of the internationally recognized standard treatment options. If conditions permit, it is recommended to conduct genetic testing as soon as possible to clarify the FLT3 status so that targeted therapy can be initiated as soon as possible in case of recurrence or insensitivity to initial treatment.

For Chinese patients, as medical insurance currently does not cover them, drug access and financial burden are key issues. It is recommended that patients obtain drugs through regular hospitals, pharmacies or certified overseas drug purchasing platforms, and use drugs in a standardized manner under the guidance of hematology specialists.

In the future, the application direction of giritinib will be more extensive. On the one hand, its combination with azacitidine, venetoclax and other drugs has entered the late clinical trial stage, and the preliminary data are encouraging; on the other hand, its potential role in bridging treatment before transplantation, maintenance treatment, and treatment-naïve patients with FLT3 mutation-positive patients is also being further studied.

With the accumulation of real-world data and the advancement of medical insurance negotiations, giritinib is expected to achieve wider clinical application and patient accessibility in China.

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References:

1. Perl AE et al. N Engl J Med. 2019;381(18):1728-1740. (ADMIRAL Research)

2. ASH Annual Meeting 2023 & 2024 Latest reports

3. FDA Gilteritinib Approval Information

4. Related documents from the Ministry of Health, Labor and Welfare of Japan and the European Medicines Agency