Wednesday Mar 29, 2023

FDA Approves Chiesi’s Lamzede® (velmanase alfa-tycv) for Alpha-Mannosidosis Treatement in USA

FDA Approves Chiesi's Lamzede® (velmanase alfa-tycv) for Alpha-Mannosidosis Treatement in USA

According to Biospace, the U.S. Food and Drug Administration (FDA) has authorized Lamzede® (velmanase alfa-tycv) for the management of alpha-mannosidosis (AM) in both adult and pediatric patients with non-central nervous system symptoms.

This news was made public by Chiesi Global Rare Diseases, a division of Chiesi Farmaceutici S.p.A., a global healthcare group with a research-focused financial model. AM is a very uncommon, progressive condition of lysosomal storage brought on by a defect in the enzyme mannosidase.

Between one in 500,000 and one in one million newborns globally are estimated to have AM disorder. AM prevents specific groupings of complex carbohydrates from being adequately broken down by the body’s cells. Several bodily functions and systems can be impacted by the accumulation of glucose.

The disease’s effects change over time and differ from individual to individual. Frequent chest and ear infections, partial deafness, unusual facial appearance, muscle spasms, musculoskeletal and joint defects, visual disorders, and mental irregularities are a few symptoms that may alter as the patient ages.

The biochemical characteristics of Lamzede, place it as a recombinant version of human alpha-mannosidase. Its function is to supplement or replace natural alpha-mannosidase enzyme that is involved in the breakdown of mannose-rich oligosaccharides and avoid their buildup in various body tissues. Lamzede’s marketing approval by the European Commission for the non-neurological treatment of symptoms in individuals with mild to moderate AM was granted to Chiesi Group in 2018.

Mark Stark, treasurer at the International Society for Mannosidosis & Related Disorders (ISMRD) said that he is thrilled to witness this milestone for the alpha-mannosidosis community. He is also a father of a son living with AM disorder.

Many such patients are hopeful that this approval gives chance to patients, caregivers, and families impacted by this devastating disease. ISMRD is expecting to collaborate with Chiesi in the future. The group is also thankful for their efforts in developing Lamzede which has very few patients across the globe. This gives patients in the U.S. a much-needed treatment option for a rare disorder.

Back to Top