EDDING announced that the applications for drug approval of lusutrombopag have been formally accepted by National Medical Products Administration in Mainland China. Lusutrombopag is indicated for the treatment of thrombocytopenia (TCP) in adult patients with chronic liver disease (CLD) who are scheduled to undergo elective invasive surgery or diagnostic manipulation.
EDDING plans to seek possible indications in Mainland China based on the previously announced successful results of the Phase III study. The results of this study are consistent with the results of the previous Phase III studies (L-PLUS 1 and L-PLUS 2) conducted overseas for lusutrombopag, which showing good safety and tolerability. The primary efficacy endpoint and key secondary endpoints of the China Phase III study are similar to the results of the two registration phase III studies of lusutrombopag in Japan and globally, and there are no new unexpected AEs compared to previous studies in the safety.
In 2019, EDDING announced an agreement with Shionogi Pharmaceutical Co., Ltd., Japan, in-licensed lusutrombopag, a thrombopoietin receptor agonist (TPO-RA) (brand name in Japan: Mulpleta®) for development and commercialization in mainland China, Hong Kong(China) and Macau(China).
Lusutrombopag is the latest generation of oral small-molecule TPO receptor agonists. Compared with the first generation, it is safer, convenient to use, and non-immunogenic and stable efficacy. And compared with other TPO-RA products, it has certain advantages, such as no potential risk of drug-drug interactions, no susceptibility of pharmacokinetics to food factors, etc. Lusutrombopag may provide CLD patients with better medical options. By acting on the transmembrane domain of the human TPO receptors, lusutrombopag activates the signal transduction pathway in the same manner as endogenous TPO to promote the production of platelets.
Mulpleta® has been approved for marketing in the Japan (2015.9), the U.S. (2018.8) and the EU (2019.2), which is indicated for the treatment of TCP in adult patients with CLD who are scheduled to undergo elective invasive surgery or diagnostic manipulation.
“We are very glad to know that the applications for drug approval of lusutrombopag were formally accepted by National Medical Products Administration (NMPA) in Mainland China,” stated James He, chief medical officer, EDDING. “Currently, most patients with thrombocytopenia caused by chronic liver disease are treated by platelet transfusion in clinical practice. However, the platelet resources in China are limited and there is a large gap between the clinical needs, so the clinical application of TPO-RA drugs is significant. We will continue working with all collaborators to bring this cross-era innovative drug into China and benefit Chinese patients.”
About Chronic Liver Disease (CLD)
Chronic liver disease, especially viral hepatitis, is an important public health problem in China. According to the data released by the Chinese Center for Disease Control and Prevention(CDC), the number of cases of viral hepatitis in Class B notifiable infectious diseases in 2020 ranked first (1,138,781), and the incidence ranked first, about 81/ 100,000[1].
CLD mainly refers to the general term for liver diseases with liver cell necrosis and slowed liver regeneration as pathological manifestations. Thrombocytopenia is a common complication in patients with CLD. According to reports, up to about 70% of patients have varying degrees of thrombocytopenia, and about 14% are complicated with severe thrombocytopenia[2].
About Thrombocytopenia （TCP）
The degree of thrombocytopenia is related to the severity of liver disease. The more severe the liver disease, the more severe the thrombocytopenia. Thrombocytopenia increases the risk of clinically significant bleeding, such as cerebral or other internal bleeding, as well as petechiae, purpura, and mucosal hemorrhage. Nowadays, there are about 460,000 people in China suffer from chronic liver disease and require elective surgery[3].
In addition, thrombocytopenia not only refers to a condition in which the number of circulating platelets in the blood is lower than the normal number, but also includes diseases in which platelets cannot function normally, mainly including idiopathic thrombocytopenic purpura (ITP), adult patients with CLD, treatment of chemotherapy-induced thrombocytopenia (CIT), thrombotic thrombocytopenic purpura, acquired thrombocytopenia, etc.