▎WuXi AppTec Content Team Report

Today (November 5), the latest announcement on the official website of the Center for Drug Evaluation (CDE) of the China National Medical Products Administration (NMPA) stated,The IND application for teprotumumab, jointly submitted by Horizon Therapeutics and Amgen, has been accepted.Public information shows that this is a"First-in-class" monoclonal antibody targeting IGF-1R, and also the first innovative therapy approved by the U.S. FDA for the treatment of thyroid eye disease.。In October 2023, Amgen completed the acquisition of Horizon Therapeutics for a total value of $27.8 billion, thereby obtaining this product.The product was also selected as a candidate for the Best Rare Disease Product Award at the 2023 Prix Galien USA Awards, known as the "Nobel Prize of the pharmaceutical industry."

Screenshot source: CDE official website

Thyroid Eye Disease (TED) is a rare, progressive, and severe autoimmune disease that threatens vision. It often occurs in patients with hyperthyroidism, caused by autoantibodies activating the insulin-like growth factor 1 receptor (IGF-1R)-mediated signaling complex in orbital cells. Symptoms include proptosis, diplopia, blurred vision, pain, and facial disfigurement. The course of thyroid eye disease is prolonged, severely affecting the patient's visual function and appearance, and can impose a heavy psychological burden on the patient.

Public data shows that multiple clinical treatment guidelines have already included antibody biologics targeting IGF-1R as a recommended treatment option, especially for TED patients with significant proptosis, where antibody biologics targeting IGF-1R can be the first choice.

Horizon Therapeutics plcDeveloped teprotumumab-trbw (Trade name:Tepezza) is an IGF-1R antibody that was first approved by the FDA in January 2020 for the treatment of thyroid eye disease.. ItCan block the activation of the IGF-1R signaling pathway mediated by related ligands such as IGF-1 or agonistic antibodies, reduce the expression of downstream inflammatory factors, alleviate inflammatory responses, and inhibit the differentiation of OFs into adipocytes or myofibroblasts, thereby reducing disease activity in TED patients and improving symptoms and signs such as proptosis, diplopia, and ocular congestion and edema.

According to the FDA's previous press release, the approval of Tepezza was based on the results of two studies (Study 1 and Study 2), which included a total of 170 patients with active thyroid eye disease who were randomly assigned to receive either Tepezza or a placebo. Among the patients treated with Tepezza, in both studies,A total of 71% and 83% of patients showed a reduction in proptosis (eye bulging) of more than 2 millimeters, compared to 20% and 10% of patients receiving placebo treatment, respectively.At the same time, Tepezza also met all secondary endpoints, including reduced double vision and improved quality of life. Based on these results, the FDA granted it Breakthrough Therapy designation, Orphan Drug status, Fast Track designation, and Priority Review status.

It is worth mentioning that in China, although teprotumumab has not yet been approved by the NMPA for clinical research, the product has already been introduced as an urgently needed imported drug in the Boao Lecheng International Medical Tourism Pilot Zone in Hainan, for the treatment of thyroid eye disease (TED). In addition, IBI311 (teprotumumab), a new biologic developed by Innovent Biologics, has already been submitted to the NMPA.Submission for Market Approval, used for the treatment of thyroid eye disease.

References:

[1] Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA) of China.Retrieved Nov 5，2024, From https://www.cde.org.cn/main/xxgk/listpage/4b5255eb0a84820cef4ca3e8b6bbe20c