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February 17, 2023February 16, 2023 - Chiesi Global Rare Diseases, (a business unit of Chiesi Farmaceutici S.p.A., an international research-focused healthcare Group, Chiesi Group), has announced that the FDA has approved Lamzede (velmanase alfa-tycv) the first and only enzyme replacement therapy for the treatment of non-central nervous system manifestations of alpha-mannosidosis (AM) in adult and pediatric patients.
AM is an ultra-rare, progressive lysosomal storage disorder caused by deficiency in the enzyme α- mannosidase, and Lamzede is indicated for the treatment of both adult and pediatric patients.
The prevalence of AM is approximately one in every 500,000 to one in every 1,000,000 babies born worldwide. AM results in the body's cells being unable to properly break down certain groups of complex sugars. The buildup of sugars can affect many of the body's organs and systems. Effects of the disease vary significantly from person to person and progress over time.
The most common adverse reactions (incidence >20%) are hypersensitivity reactions including anaphylaxis, nasopharyngitis, pyrexia, headache, and arthralgia.
Lamzede may cause embryo-fetal harm when administered to a pregnant female. A negative pregnancy status must be verified in females of reproductive potential prior to initiating the medication. Patient should be advised to use effective contraception during treatment and for 14 days after the last dose of Lamzede.