A New Treatment For Alpha-Mannosidosis Just Got Authorized By The EMA

According to an announcement from ema.europa.eu, the European Medicines Agency (EMA) recently granted market authorization for a new treatment for alpha-mannosidosis. The new drug is an enzyme replacement therapy called Lamzede. The treatment is approved for long term usage by both children and adults who have mild to moderate forms of the condition.
Alpha-mannosidosis is a lysosomal storage disorder in which the activity of the enzyme alpha-d-mannosidase is deficient. This enzyme normally breaks down the sugars from glycoproteins, but in an affected person, these sugars build up in the body and inhibit normal cell function. This disorder is caused by a genetic mutation. This condition can vary significantly in the beginning of onset and severity. In the most severe cases, symptoms occur within the first few months of life. These include intellectual disability, skeletal abnormality, enlargement of the liver and spleen, hearing loss, and lung infections. People with alpha-mannosidosis often exhibit distinctive facial features, such as a wide mouth, prominent forehead, and a flat nasal bridge. Prognosis depends on the severity of the disorder; people with severe alpha-mannosidosis may not survive their childhood, but people with milder cases can live well into their adult lives. To learn more about this condition, click here.

Lamzede represents the first attempt at a treatment that aims to treat the cause of alpha-mannosidosis. Previously, treatment for the disorder involved reducing the severity of disease symptoms and complications. As an enzyme replacement therapy, Lamzede is a recombinant form of alpha mannosidase that will serve as a replacement for the deficient amount in patients. The treatment is shown to prevent further progression of the condition, but since it is unable to cross the blood-brain barrier, it can not work against the disease’s neurological effects.

While the effectiveness of Lamzede was proven during clinical trials, the rarity of the disease meant that the Committee for Medicinal Products for Human Use agreed that it would be impossible to obtain credible data on safety and efficacy under normal usage conditions. Thus, the marketing authorization was granted under “exceptional circumstances” which will allow patients to access treatments that could not be sufficiently tested under the normal standards. The EMA also granted Lamzede orphan designation.

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