Chiesi Global Rare Diseases, a business unit of Chiesi Farmaceutici S.p.A., an international research-focused healthcare Group (Chiesi Group), announced that the U.S. FDA has approved Lamzede® (velmanase alfa-tycv) 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.
“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. Lamzede is designed to provide an exogenous source of the α-mannosidase enzyme and we look forward to offering this medicine to patients in the United States who are eagerly awaiting a treatment option.”
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 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.
