Glibenclamide oral suspension: Suitable and effective in patients with neonatal diabetes复制标题

格列本脲口服混悬液对新生儿糖尿病患者的治疗效果

Neonatal diabetes (NDM) was treated in the same way as type 1 diabetes until elucidation of the genetic basis of with the disease was shown to involve activating mutations in KCNJ11 or ABCC81. This discovery led to the first example of precision medicine in diabetes and to the radically different, specific and successful therapeutic approach consisting in giving oral sulfonylureas (glibenclamide, also known as glyburide) instead of insulin injections. Patients carrying these mutations can produce insulin but have an insufficient insulin response to glucose. In addition to providing therapeutic benefits, oral glibenclamide treatment is more convenient than insulin injections, eliminates the pain due to injections, and provides greater flexibility, thereby allowing optimization of the dosage regimen and much better glycemic control. The better glycemic control is associated with improvements indyspraxia, supporting the early initiation of sulfonylureas (SU) therapy.Precision medicine in pediatrics requires suitable drug preparation allowing the administration of the lowest possible dosage that provides optimal glycemic control and neuropsychological improvements. Until recently, glibenclamide was available only as tablets, which had to be crushed and mixed with water for administration to neonates and infants. Even when prepared by hospital pharmacies, crushed tablets in water lack reliability as they have no microbiological stability and vary in concentration over time due to gradual sedimentation of the glibenclamide, which has limited solubility in water. AMMTeK has developed a paediatric glibenclamide (named glyburide in the US) oral suspension (AMGLIDIA®), which has been designated as an orphan drug for the treatment of NDM in europe and the US. Compared to crush tablets the suspension assures more consistent pharmacokinetics and could be more acceptable for children.

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