FDA approves Lamzede for non-CNS alpha-mannosidosis in adults, children

The FDA has approved Lamzede, an enzyme replacement therapy for non-central nervous system manifestations of alpha-mannosidosis in adult and pediatric patients, Chiesi Global Rare Diseases announced in a press release.

Velmanase alfa-tycv (Lamzede, Chiesi) is a recombinant form of human alpha-mannosidase designed to supplement the body’s natural enzyme, which prevents accumulation of mannose-rich oligosaccharides in various tissues in the body.

The treatment will be the first enzyme replacement therapy available to U.S. adults and children with this rare, progressive lysosomal storage disorder, which is prevalent in one in every 500,000 to 1 million infants globally, according to the release.

The FDA approval is a “major milestone for people living with alpha-mannosidosis,” Giacomo Chiesi, head of Chiesi Global Rare Diseases, said in the release. “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.

“We look forward to offering this medicine to patients in the United States who are eagerly awaiting a treatment option.”