The first treatment for thyroid ophthalmopathy! Horizon's IGF-1R targeted monoclonal antibody tepezza (teprotumumab) has been approved by the US FDA!

January 22, 2020 / BIOON / -- horizon Pharma is a pharmaceutical company focusing on the development and commercialization of innovative drugs to meet the treatment needs of rare and rheumatic diseases Recently, the company announced that the US Food and Drug Administration (FDA) has approved tepezza (tepromumab trbw) for the treatment of thyroid ophthalmopathy (TED) This approval is more than one month ahead of the target date of PDUFA on March 8, 2020 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 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 In December 2019, teprotumumab obtained the full vote (12-0) of FDA's dermatology and ophthalmic drug advisory committee (dodac) "Today is a great day for the Ted patient community," said Timothy Walbert, President and CEO of horizon Ted is a rare, eye threatening disease that has not previously been approved by the FDA Tepezza's approval is significant and will change the treatment model of Ted and provide new hope for patients with this terrible disease " This approval is based on positive data from phase II clinical study and confirmatory phase III clinical study optic (nct03298867) The top line results of phase III study published in March 2019 showed that the study reached the main end point Compared with the placebo group, the patients in the teprotumab treatment group showed significant improvement in exophthalmos or bulging: 82.9% of the patients in the teprotumab treatment group reached the main end point of exophthalmos or bulging reduction of 2mm or more, while only 9.5% in the placebo group, with statistically significant difference (P < 0.05) .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, teprotumumab 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 teprotumab treatment resulted in clinically significant and highly statistically significant reductions in exophthalmos and active Ted symptoms (pain, swelling, redness, inflammation) Thyroid ophthalmopathy (TED) (picture source: Mister sign Info) thyroid ophthalmopathy (TED) is a progressive, 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 Original source: FDA approvals tepezza (TM) (teprotumab trbw) for the treatment of tyre eye disease (TED)