Sorafenib: the only targeted thyroid cancer drug available in China

  • Launched in China in 2017 (trade name: Doxorubicin) for the treatment of locally recurrent or metastatic, iodine-refractory differentiated thyroid cancer, and included in health insurance.

Sorafenib (sorafenib) is a tyrosine kinase inhibitors (TKI) class drug that has multiple targets, including vascular endothelial growth factor receptors (VEGFR) , platelet-derived growth factor receptor (PDGFR), common RET/PTC subtypes, stem cell factor receptor (c-Kit), and BRAF genes. Current approved indications include refractory thyroid cancer.

Application: iodine-refractory advanced DTC

The American Thyroid Association (ATA) guidelines state that patients with metastatic differentiated thyroid cancer (DTC) that has not responded to conventional therapy (tumors larger than 1 to 2 cm and growing at least 20% per year), or who develop overt symptoms, should be treated with an iodine-refractory gene. In 2013, sorafenib was approved in the United States for the treatment of advanced DTC that no longer responds to radioactive iodine (RAI) therapy.

TKI-like drugs are recommended for patients with metastatic DTC who are unable to participate in clinical trials.

In 2017, sorafenib (trade name: doxorubicin) was approved in China for the treatment of locally relapsed or metastatic, progressive, iodine-refractory DTC (radioactive iodine refractory-DTC, RR-DTC) and has been entered into Medicare.

Some early studies have shown that sorafenib prolongs survival in patients with DTC. In a phase III clinical study in the United States, 417 patients with advanced RR-DTC treated with sorafenib achieved a median progression-free survival of 10.8 months, meaning that half of these very refractory patients achieved nearly 1 year of disease control, compared with 5.8 months in the placebo group.

Domestic and international studies: exploring a wider range of indications, better regimens

Sorafenib is also being explored in other types of rare thyroid cancer, and has shown some benefit in both undifferentiated thyroid cancer (ATC) and progressive or metastatic medullary thyroid carcinoma (MTC).

In addition, studies of sorafenib for preoperative treatment of DTC and its combination with everolimus and temsirolimus for refractory DTC are ongoing and have shown good results (Tables 1 and 2).

Table 1: Foreign studies of sorafenib in thyroid cancer

Clinical trial number

Study Subjects

Test state

Purpose of the study

NCT01141309

Thyroid cancer 18 years and older

On-going; no longer recruiting

Efficacy of everolimus + sorafenib in the treatment of thyroid cancer

NCT01263951

Metastatic DTC

On-going; no longer recruiting

Comparing the efficacy of everolimus + sorafenib versus sorafenib alone for advanced thyroid cancer

NCT01025453

Radioiodine-refractory thyroid cancer

In progress; no longer recruiting

Efficacy of Sorafenib + Tesilomox for Iodine-Refractory Thyroid Cancer

NCT03565536

ATC

Recruiting

Sorafenib for preoperative adjuvant therapy in ATC

(Source: clinicaltrials.gov)

Clinical trial number

Table 2: Domestic studies of sorafenib in thyroid cancer

Study Subjects

Test state

Purpose of the study

CTR20130038

Thyroid cancer

In progress; no longer recruiting

Efficacy of Sorafenib for Thyroid Cancer

(Source: Drug Clinical Trial Registration and Information Disclosure Platform)

Summary

Sorafenib prolongs survival in patients with iodine-refractory DTC and has manageable side effects. As research progresses, it is likely that more and more sorafenib-based combination therapy regimens will emerge to benefit more patients.