The first treatment for thyroid ophthalmopathy! Horizon's IGF-1R targeted monoclonal antibody teprotumumab was approved by the US FDA Expert Committee!

December 29, 2019 / 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 U.S Food and Drug Administration (FDA) dermatology and ophthalmic drug advisory committee (dodac) unanimously approved and supported the potential benefits of teprotumumab in the treatment of active thyroid ophthalmopathy (TED) greater than the risks At present, teprotumab's biologicals licensing application (BLA) is under FDA's priority review (PR), and its PDUFA target date is March 8, 2020 Dodac's opinion is not legally binding on FDA, but FDA will generally adopt its opinion when reviewing If approved, teprotumumab will be the first drug to treat active Ted Previously, the FDA has granted teprotumumab priority review, breakthrough drug qualification, orphan drug qualification and fast track qualification for active Ted Priority review (PR) is a new drug review channel established by FDA, which grants drugs that can provide significant improvement in the treatment, diagnosis or prevention of diseases In the priority review process, FDA will complete the drug application review within 6 months, compared with 10 months in the standard review Timothy Walbert, President and CEO of horizon, said: "the unanimous approval of dodac marks an important step in bringing the first FDA approved treatment to Ted patients The comprehensive data on the efficacy and safety of teprotumumab in the treatment of active Ted provided by us proves the extraordinary efforts made by the doctors we worked with, the Ted patients who participated in the research project, and the R & D team of horizon We believe that teprotumab has the potential to address the important unmet medical needs of these patients We look forward to working closely with teprotumab in the FDA review process to bring this innovative therapy to patients as soon as possible " The BLA of teprotumab is based on the positive data of the confirmatory phase III clinical study optic (nct03298867) and phase II clinical study The top line results of the phase III study published in March this year showed that the study reached the main end point, and patients in the teprotumumab treatment group showed significant improvement in exophthalmos or bulging compared with those in the placebo group Results: 82.9% of the patients in the teprotumab group reached the main end point of exophthalmos or reduction of bulge by 2 mm or more, while that in the placebo group was only 9.5% (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, 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 Teprotumab is an all human IgG1 monoclonal antibody targeting insulin-like growth factor-1 receptor (IGF-1R), which is 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 If approved, teprotumumab will provide clinicians with the first drug to reduce exophthalmos during active Ted, in addition to treating other painful symptoms Original source: FDA advisory committee votes unanimity to support the use of teprotumab for the treatment of gyroid eye disease (TED)