A new chance for the treatment of neonatal diabetes mellitus: CHMP recommends granting the marketing authorization for Amglidia

A new chance for the treatment of neonatal diabetes mellitus: CHMP recommends granting the marketing authorization for Amglidia

In February 2018, the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has recommended granting a marketing authorization in the European Union for Amglidia, a medicine indicated for the treatment of neonatal diabetes mellitus (NDM). NDM is a rare monogenic form of diabetes that occurs in the first 6 to 12 months of life and is caused by different gene mutations. In about half of patients affected by NMD, the condition is lifelong, in the rest of the cases it is transient or temporary and it can disappear during infancy but reappear later in life. Symptoms of NMD include frequent urination, rapid breathing and dehydratation, but it can cause more severe complications due to the lack of insulin production, high blood sugar levels and the consequent risk of ketoacidosis. Amglidia is a new formulation of glibenclamide, a medicine already authorized for treating type 2 diabetes, specifically developed for use in newborns, toddlers and children with neonatal diabetes. It works on insulin-producing cells in the pancreas by attaching to an ATP-sensitive potassium (KATP) channel, which controls the release of insulin. In many newborn babies with neonatal diabetes, the cells in the pancreas produce insulin but they are not able to release it into the blood because their gene mutations lead to dysfunctional KATP channels. Glibenclamide’s effect on the KATP channel restores the cells’ ability to release insulin into the blood. The new oral formulation of this drug represents a new opportunity in the NDM treatment that could avoid the risk of error administration and under- or over- dosing, due to the current method of administration of off-label commercially- available glibenclamide. 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, increasing the possibility of administration errors. The NEOGLI study demonstrated the effectiveness of Amglidia, showing that glycaemic control remained stable after switching from crushed tablets to oral suspension. Due to the extreme rarity of the disease, there were only 10 patients included in the NEOGLI study. NMD, in fact, is a very rare disease and Amglidia was granted an orphan designation in January 2016. This orphan designation will now be reviewed by EMA’s Committee for Orphan Medicinal Products (COMP) to determine whether the information available to date allows maintaining Amglidia’s orphan status and granting this medicine ten years of market exclusivity. The opinion adopted by the CHMP at its February 2018 meeting is an intermediate step on Amglidia’s path to patient access. The CHMP opinion will now be sent to the European Commission for the adoption of a decision on an EU-wide marketing authorisation. Once a marketing authorisation has been granted, decisions about price and reimbursement will take place at the level of each Member State, taking into account the potential role/use of this medicine in the context of the national health system of that country.

More information are available here.

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