Chiesi Global Rare Diseases, a business unit of Italy-based pharmaceutical company Chiesi Farmaceutici S.p.A., announced on Thursday that it has received approval from the US Food and Drug Administration (FDA) for Lamzede (velmanase alfa-tycv) to treat non-central nervous system manifestations of alpha-mannosidosis (AM) in adult and paediatric patients.

AM is an ultra-rare, progressive lysosomal storage disorder caused by deficiency in the enzyme alpha-mannosidase.

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 authorisation from the European Commission for Lamzede for the treatment of non-neurological manifestations in patients with mild to moderate AM.

Giacomo Chiesi, head of Chiesi Global Rare Diseases, said: "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.

"Alpha-mannosidosis presents with a variety of symptoms including impaired hearing, speech and mobility that progress from childhood into adulthood and is often under-recognised, causing some patients to be left undiagnosed or untreated. Lamzede is designed to provide an exogenous source of the alpha-mannosidase enzyme and we look forward to offering this medicine to patients in the United States who are eagerly awaiting a treatment option."