Jan 23, 2020
Tepezza (teprotumumab-trbw) is the first-ever treatment approved for thyroid eye is a progressive autoimmune disorder hampering the ability of vision. The drug was approved under Priority Review and had received Orphan Drug, Fast Track and Breakthrough Therapy designations from the FDA.
Thyroid eye is a rare disease; however, up to 50 per cent of patients with Graves’ disease, the most common autoimmune disorder, are at the risk of developing TED.
Tepezza is a fully human monoclonal antibody (mAb) and a targeted inhibitor of the insulin-like growth factor-1 receptor (IGF-1R). It is administered once every three weeks for a total of eight infusions. The clinical trials Phase II and III showed improvements in patients as early as six weeks, as well as continued improvement across the entire 24-week course of treatment.
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The study, undertaken at the Lewis Katz School of Medicine at Temple, points towards the use of pharmacological chaperones, small drug molecules, to get rid of clumps of dysfunctional proteins. The study revealed a new approach to hamper the process of damaging the neurons in the brain by restoring the levels of VPS35, a sorting molecule, that eliminates the clump of old proteins.
They can offer a new approach to deal with neurological conditions, as they are relatively inexpensive. Moreover, the drugs do not block an enzyme or a receptor but target a cellular mechanism, reducing the risk of significant side effects.
Treatment with KTX-0200 has helped with sustained weight loss of 14% in chronic diet-induced obesity rodent models. Moreover, it also helped in improving markers of health such as glucose tolerance and insulin sensitivity.
The drug, KTX-0200, an oral small molecule anti-obesity agent, has demonstrated the potential cure of treating diabetes. Moreover, it might help in improving liver health.
The new research would help curb the increasing burden of obesity, and other metabolic diseases, including type 2 diabetes, NASH, and cardiovascular diseases.
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