LAMZEDE (velmanase alpha), enzyme

Moderate clinical benefit in non-neurological manifestations of mild to moderate alpha-mannosidosis and minor clinical added value in terms of stabilisation of the disease.   

• LAMZEDE has been granted a marketing authorisation for the enzyme replacement therapy of non-neurological manifestations in patients suffering from mild to moderate alpha-mannosidosis.

• Its efficacy is superior to that of the placebo in terms of a biological endpoint, i.e. the reduction of serum oligosaccharide concentration after one year of treatment.

• The results for various clinical endpoints suggest a stabilisation in disease progression with LAMZEDE compared to a deterioration observed with the placebo.

• Uncertainties remain concerning the long-term efficacy and safety of this medicinal product administered by weekly intravenous infusion.

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