ESPE Abstracts (2018) 89 RFC12.6

AMGLIDIA, a Suspension of Glibenclamide for Patients with Neonatal Diabetes, Long Term Data on Efficiency and Tolerance

Jacques Beltrand, Candice Meyzer, Sandra Colas, Michaela Semeraro, Cécile Godot, Jean-Marc Treluyer, Caroline Elie & Michel Polak


Hôpital Universitaire Necker Enfants Malades, AP-HP, Faculté de médecine Paris Descartes, Paris, France


Background: Glibenclamide has proven to be efficient for patients with neonatal diabetes owing to potassium channel mutations. We developed a suspension of glibenclamide (EMA CHMP Authorization February 2018) fitting recommendations of drug administration to allow a precise dosage. We reported it to be practical, efficient and well tolerated after 3 months of use.

Objective: To determine long term efficiency and tolerance of a new suspension of glibenclamide AMGLIDIA.

Method: Patients where switched from crushed tablets to suspension and prospectively followed-up. Efficiency and tolerance were recorded (Dosage, number of preprandial hyperglycaemia, number of hypoglycaemia, adverse events, HbA1C). Adverse events were recorded.

Results: Five patients (three boys) with neonatal diabetes owned to KCNJ11 mutation were treated and prospectively followed-up. Suspension was introduced as a very young age and some months after sulfonylureas introduction. Indeed, median age at suspension introduction was 1.5 years of age (0.47 to 1.7 years of age). Treatment duration with AMGLIDIA suspension ranged from 20 to 34 months. Median dosage at introduction 0.1 mg/kg per d (0.06 to 0.26 mg/kg per d) and didn’t change significantly throughout the follow-up as median dosage was 0.08 mg/kg per d (0.03 to 0.27 mg/kg per d). Metabolic control remained excellent throughout the follow-up: HbA1C at initiation: 6.7% (5.1 to 7.9%), HbA1C at last visit: 5.5% (5.5 to 6.5%). During the month before last visit, seven hyperglycaemia > 250 mg/dl was recorded in one patient and five hypoglycaemias (2 in on and 3 in another one). Safety record was excellent: 1 asymptomatic hypoglycaemia below 54 mg/dl was recorded another hypoglycaemia with neurological symptoms in the context of acute viral gastroenteritis. No other adverse event was recorded.

Conclusion: Glibenclamide suspension AMGLIDA allows an excellent long term metabolic control and is very well tolerated. (ClinicalTrial.gov NCT02375828).

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