FDA approves alpha-mannosidosis drug

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FDA

Chiesi Global Rare Diseases, a business unit of Chiesi Farmaceutici S.p.A., an international research-focused healthcare Group (Chiesi Group), says the U.S. Food and Drug Administration (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.”

Alpha-mannosidosis

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.

About Chiesi’s Lamzede

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.

“We are thrilled to witness this milestone for the alpha-mannosidosis community,” said Mark Stark, treasurer at the International Society for Mannosidosis & Related Disorders (ISMRD) and father of a son living with AM. 

“I have watched first-hand how alpha-mannosidosis can progress and impact daily life with my son and this approval gives hope to patients, caregivers and families impacted by this devastating disease. ISMRD is excited to continue to collaborate with Chiesi and we are thankful for their efforts to develop Lamzede to give patients in the U.S. a much-needed treatment option.”

The company will also be presenting eight abstracts at the 19th annual WORLD Symposium research meeting from February 22 to 26, 2023 in Orlando, Florida. The company will also support satellite symposia focused on lysosomal diseases including Fabry disease and alpha-mannosidosis.

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