Approves Enzyme Replacement Therapy for Rare Disease

The FDA has approved Lamzede (velmanase alfa-tycv) to treat adults and children with alpha-mannosidosis, a rare genetic condition where the body is unable to properly break down certain complex sugars in cells. The buildup of sugars can affect many of the body's organs and systems. Alpha-mannosidosis is an ultra-rare, progressive lysosomal storage disorder caused by deficiency in the enzyme α-mannosidase. Alpha-mannosidosis affects about 1 in every 500,000 people worldwide.

Developed by Chiesi Global Rare Diseases, Lamzede is a recombinant form of the human alpha-mannosidase and indicated to address the non-central nervous system manifestations of the disease. Company officials planning to make Lamzeda available in the first half of this year. The company isn’t discussing the cost, a spokesperson said.

Once available, physicians and patients can access Lamzede the company’s support program Chiesi Total Care. “We plan to work closely with U.S. payers to make Lamzede available to as many eligible patients as possible,” the spokesperson said.

“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. Lamzede is designed to provide an exogenous source of the α-mannosidase enzyme,” Giacomo Chiesi, head of Chiesi Global Rare Diseases, said in a press release.

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 alpha-mannosidosis.