The European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) has recommended granting approval for a new oral suspension formulation of the sulfonylurea glibenclamide (Amglidia, Ammtek) for the treatment of newborns and infants with neonatal diabetes.
Neonatal diabetes is an extremely rare form of diabetes diagnosed in the first 6 months of life. It is life-threatening because of the symptoms caused by hyperglycemia and risk of ketoacidosis.
Separately, CHMP has also granted an extension of indication to anakinra (Kineret, Sobi) for the treatment of Still’s disease, a rare disease that causes joint inflammation, as well as rash and fever, in children and adults.
The new indication for Still’s disease includes systemic juvenile idiopathic arthritis (JIA) and adult-onset Still’s disease, which are debilitating and difficult-to-treat disorders with few approved treatment options.
R ecommendation for Approval of New Oral Suspension of Glibenclamide
Glibenclamide is already authorized for use in type 2 diabetes, but currently medical staff, or parents, have to crush tablets into small pieces for adults and give to infants by mixing with water and administering with an oral syringe. This can lead to errors in dosing.
The benefits of Amglidia are supported by data published in the literature as well as the results from a bioavailability study, and a trial called NEOGLI conducted in 10 patients, EMA notes. The results of NEOGLI showed that glycemic control remained stable after switching from crushed glibenclamide tablets to oral suspension.
Amglidia will be available as an oral suspension (0.6 and 6 mg/mL of glibenclamide) and it is recommended that the medication be started by a physician experienced in the treatment of patients with very early onset diabetes.
Amglidia was granted orphan drug status in January 2016.
Glibenclamide as the product Daonil has been authorized in the European Union since January 1969, EMA notes, and because Amglidia is a hybrid medicine of Daonil, the application can rely in part on the results of preclinical tests and clinical trials of the reference product and in part on new data, the agency says.
Another Option for Still’s Disease
Anakinra is an immunosuppressant, which blocks interleukin 1, produced in high levels in patients with rheumatoid arthritis and Still’s disease (systemic JIA), causing joint inflammation, joint damage, and systemic features such as fever, skin rash, and inflammation of internal organs.
Currently, most patients with Still’s disease are initially treated with anti-inflammatory drugs, including nonsteroidal anti-inflammatory drugs (NSAIDs) and glucocorticoids, which can be effective at controlling the disease and its symptoms.
However, high doses of steroids are often needed to control inflammation, which can lead to a range of side effects. Other treatment options include monoclonal antibodies that are used as second-line treatment. There is still an unmet medical need for authorized efficient treatment for the disease.
EMA notes that Still’s disease is the most severe form of arthritis in children.
The recommendation for an additional indication for Kineret is for adults, adolescents, children, and infants aged 8 months and older with a body weight of 10 kg or above for the treatment of Still’s disease, including systemic JIA and adult-onset Still’s disease, with active systemic features of moderate-to-high disease activity, or in patients with continued disease activity after treatment with NSAIDs or glucocorticoids.
Kineret can be given as monotherapy or in combination with other anti-inflammatory drugs and disease-modifying antirheumatic drugs.
Recognizing the potential of anakinra to treat children with Still’s disease, it was agreed to address this pediatric need, and availability of the treatment option for infants, children, and adolescents was facilitated through discussions between the marketing authorization holder and EMA’s pediatric committee on a pediatric investigation plan (PIP).
The CHMP’s positive opinion is based on data from clinical trials (two studies were included as part of the PIP) as well as the scientific literature and meta-analyses of published data. Overall, the evaluation of the medicine is based on 442 patients with Still’s disease (245 pediatric and 197 adult).
It shows the efficacy of anakinra in both pediatric and adult patients with Still’s disease, with the majority of patients achieving remission as well as an improvement of the signs and symptoms associated with the condition.
Safety data from the clinical trials and published literature in Still’s disease, together with substantial safety data from post-marketing in both Still’s disease and other indications, showed that the most common adverse events were injection site reactions, as well as fever, rash, and headache, says EMA.
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