FDA last week approved velmanase alfa (Lamzede), the first enzyme replacement therapy approved in the U.S. for the treatment of non-central nervous system manifestations of alpha-mannosidosis, a rare genetic condition characterized by the lack of the alpha-mannosidase enzyme in the body. Velmanase alfa restores normal cellular activity by acting in the same way as the alpha-mannosidase enzyme. The symptoms of alpha-mannosidosis vary, but often include mild-to-moderate intellectual disability, hearing loss, weakened immune system, distinctive facial features (e.g., a large head, prominent forehead, and protruding jaw), skeletal abnormalities, and muscle weakness. Alpha-mannosidosis affects about 1 in every 500,000 people worldwide. The most common adverse reactions to velmanase alfa are hypersensitivity reactions, including anaphylaxis.
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