European regulators have green-lighted Chiesi’s Lamzede as the first long-term enzyme replacement therapy to treat non-neurological manifestations in patients with mild to moderate alpha-mannosidosis.
The condition is a rare inherited enzyme disorder that causes cell damage through build-up of mannose-rich oligosaccharides in many organs and tissues of the body.
Patients with the disease can have intellectual disability, liver or spleen enlargement, distinctive facial features and skeletal abnormalities. Those with early onset severe and rapid progressive disease often do not survive beyond childhood, while those with milder forms survive into adult life.
There is currently no cure for this disease. Lamzede (velmanase alfa) is designed to replace the function of the deficient enzyme in the body, to normalise oligosaccharide levels and prevent disease progression.
However, the therapy does not cross the blood-brain-barrier and is thus not expected to have an effect on the neurological aspects of the disease.
Marketing approval was granted under ‘exceptional circumstances according to the EU legislation, which aim to enable treatment of extremely rare disorders for which traditional large-scale clinical studies are not feasible; Lamzede was investigated in 34 patients, both children and adults.
“Lamzede is the first approved treatment with disease modifying potential for alpha-mannosidosis and therefore, our next goal is to make this treatment available to patients in Europe as soon as possible,” comments Alessandro Chiesi, Region Europe head, Chiesi Group.
“The marketing authorisation of Lamzede in the EU is a significant stepping stone for patients suffering from this devastating disease.”