RARE Daily

FDA Approves Chiesi’s Lamzede for the Treatment of Alpha-Mannosidosis

February 17, 2023

Rare Daily Staff

The U.S. Food and Drug Administration has approved Chiesi Global Rare Diseases’ Lamzede for the treatment of non-central nervous system manifestations of alpha-mannosidosis in adult and pediatric patients.

“Today’s approval of Lamzede represents a major milestone for people living with alpha-mannosidosis. Lamzede is the first and only enzyme replacement therapy approved for alpha-mannosidosis in the United States, an achievement based on years of clinical development, as well as the dedication of our employees, clinicians, patients, and their families,” said Giacomo Chiesi, head of Chiesi Global Rare Diseases. “Alpha-mannosidosis presents with a variety of symptoms including impaired hearing, speech and mobility that progress from childhood into adulthood and is often under-recognized, causing some patients to be left undiagnosed or untreated.”

Alpha-mannosidosis (AM) is an ultra-rare, progressive lysosomal storage disorder caused by deficiency in the enzyme α-mannosidase. 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. Symptoms may change as a patient gets older and can include recurrent chest and ear infections, hearing loss, distinctive facial features, muscle weakness, skeletal and joint abnormalities, visual abnormalities, and cognitive abnormalities.

Lamzede (velmanase alfa) is a recombinant form of human alpha-mannosidase intended to provide or supplement natural alpha-mannosidase, an enzyme that is involved in the degradation of mannose–rich oligosaccharides to prevent their accumulation in various tissues in the body. In 2018, Chiesi Group received marketing authorization from the European Commission for Lamzede for the treatment of non-neurological manifestations in patients with mild to moderate AM.

Photo: Giacomo Chiesi, head of Chiesi Global Rare Diseases


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