The first treatment for thyroid ophthalmopathy! IGF-1R targeted monoclonal antibody tepezza (teprotumumab) in phase III clinical significantly improved exophthalmos and diplopia!

Thyroid ophthalmopathy (TED) (photo source: Mister sign Info) January 28, 2020 / Bio Valley bio on / -- horizon Pharma is a pharmaceutical company focusing on the development and commercialization of innovative drugs to meet the treatment needs of rare diseases and rheumatic diseases Recently, the company announced that the results of the evaluation of tepezza (tepromumab trbw) phase III opt IC clinical trial for the treatment of thyroid ophthalmopathy (TED) have been published in the New England Journal of Medicine (NEJM) The article title is: tepromumab for the treatment of active thaloid eye disease The study is part of the largest clinical project in Ted, and data show that tepezza shows improved exophthalmos and diplopia in Ted patients compared to placebo Ted is a serious, progressive and rare autoimmune disease threatening vision, which is related to exophthalmos, diplopia, blurred vision, pain, inflammation and facial defects Tepezza is an all human monoclonal antibody and insulin-like growth factor-1 receptor (IGF-1R) targeting preparation It was approved by the U.S Food and Drug Administration (FDA) on January 21, 2020, and used in the treatment of Ted The drug is injected every three weeks, a total of 8 times It is worth mentioning that tepezza is the first and only drug approved by FDA to treat Ted Previously, the FDA has granted teprotumumab priority review, breakthrough drug qualification, orphan drug qualification and fast track qualification for active Ted The optic study compared the efficacy and safety of tepezza with placebo, which was administered eight times a week The results showed that the primary end point and all secondary end points were reached In terms of primary endpoint, at the 24th week of treatment, the patients in tepezza group showed significant improvement in exophthalmos or bulging compared with placebo group: 83% of the patients in tepezza group reached the primary endpoint of 2 mm or more reduction in exophthalmos or bulging, while only 10% of the patients in placebo group, with statistically significant difference (P < 0.001) Proptosis is the main cause of Ted In addition, the study also reached all secondary endpoints, including reduced diplopia, improved quality of life, and reduced clinical activity scores In this study, tepezza was well tolerated, most of the adverse events were mild or moderate, which could be controlled and resolved during or after treatment Data from the phase II study showed that tepezza treatment resulted in clinically significant and highly statistically significant reductions in exophthalmos and active Ted symptoms (pain, swelling, redness, inflammation) "Ted is a rare and devastating autoimmune disease that has not been adequately treated," said Dr Raymond Douglas, CO lead researcher of the optic trial and director of the orbital and thyroid diseases program at cedars Sinai Medical Center Patients must wait several years for the disease to become inactive before they can receive surgery These operations are not only complex, but often fail to fully restore vision or appearance In the optic trial, we found statistically significant improvements in key symptoms, including exophthalmos and diplopia, in the first patient assessment after 6 weeks of treatment, and these improvements continued over the 24 week treatment period Tepezza has just been approved by the US FDA This approval is of great significance The drug has the potential to change the treatment mode of Ted " Thyroid ophthalmopathy (TED) is a progressive and aging autoimmune disease, with limited active disease window, which can be treated without surgical intervention during this period Although Ted often occurs in patients with hyperthyroidism or Graves' disease, it is a unique disease caused by the activation of igf-1r-mediated signal complexes in orbital cells by autoantigens This will lead to a series of negative effects, resulting in long-term, irreversible damage Active Ted lasted for 3 years, characterized by inflammation and tissue expansion With the development of Ted, it can cause serious damage, including eye protrusion (exophthalmos), strabismus (dislocation of eyeball), diplopia (two vision), and in some cases, blindness At present, patients must go through active Ted until the disease becomes inactive (which usually leaves permanent and vision impaired consequences), before they can have complex and expensive surgery, but the surgery may never be able to restore vision or appearance Ted patients often experience long-term functional, psychological and economic burdens, including being unable to work and engaging in activities of daily life Tepromumab, the active drug component of tepezza, is an all human IgG1 monoclonal antibody targeting insulin-like growth factor-1 receptor (IGF-1R), which has been developed for the treatment of moderate to severe thyroid ophthalmopathy (TED), which is usually associated with Graves' disease (hyperthyroidism) In the optic study, patients treated with teprotumab experienced an unprecedented reduction in exophthalmos, which can only be treated surgically after the end of active disease Horizon expects to launch tepezza in the U.S market in the next few weeks, which will provide clinicians with the first drug to reduce exophthalmos during active Ted, in addition to treating other painful symptoms The company will also conduct a post market study to assess the safety of tepezza in a larger patient population, as discussed at the dodac meeting on December 13, 2019 The study will also assess the relationship between the recovery rate and the length of treatment The New England Journal of Medicine publishes comprehensive data from phase 3 clinical trial (optional) of tepezza Gamma (teprotumumab-trbw) for Thyroid Eye Disease