CHMP recommends Amglidia to treat neonatal diabetes.-Ammtek

The European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) recommends the approval of Amglidia from Ammtek as an oral suspension (0.6 mg / ml and 6 mg / ml) for the treatment of neonatal diabetes.

The active substance is glibenclamide, a sulfonylurea, which stimulates insulin secretion from pancreatic beta-cells by inhibiting ATP-sensitive potassium channels. The benefit of the drug is its ability to improve glycemic control. The most common side effects are hypoglycaemia, transient diarrhea and abdominal pain.

Amglidia is a hybrid drug from Daonil, approved in the EU since 1 January 1969. Amglidia contains the same active substance as Daonil but is approved for a different indication and is available in a different formulation and strength. Studies have demonstrated the satisfactory quality and relative bioavailability of Amglidia.

The recommendation for approval is based on published data from the research literature as well as data from a bioavailability study and the NEOGLI study. Due to the extreme rarity of the disease, only 10 patients were included in the NEOGLI study. It was found that the glycemic control remained stable after switching from crushed tablets to oral suspension.

Comment: Currently, to treat neonatal diabetes, nursing staff under medical prescription, or the parents at home, administer insulin or off-label commercially-available glibenclamide tablets licensed for adults only. To make the products suitable for newborns and children, the tablets are crushed into small pieces and given to the infant by mixing the fragments with a small amount of water; the mixture is then administered with an oral syringe. This practice can cause errors in the administration, potentially leading to a risk of under- or over-dosing. Amglidia’s formulation is meant to allow a more accurate dosing of glibenclamide, addressing a clear unmet medical need. Additionally, patients treated with Amglidia may not need to be treated with insulin or may need a smaller dosage.