Velmanase alfa-tycv (Lamzede) is approved as the first and only enzyme replacement therapy for non-central nervous system manifestations of alpha-mannosidosis, an extremely rare, progressive, lysosomal storage disorder.
Velmanase alfa-tycv (Lamzede) became the first and only enzyme replacement therapy approved by the US Food and Drug Administration (FDA) for treating non-central nervous system manifestation of alpha-mannosidosis (AM) in the US. The therapy was indicated for both adult and pediatric patients, according to an announcement from Chiesi Global Rare Diseases, of Chiesi Group.1
The FDA decision was pivotal for individuals with alpha-mannosidosis because of the condition’s status as an ultra-rare, progressive lysosomal storage disorder caused by a deficiency in the enzyme α-mannosidase. Estimates mark the prevalence of alpha-mannosidosis at one in every 500,000, to one in every 1 million babies born worldwide. It’s not uncommon for individuals with the condition to go untreated or undiagnosed.
"We are thrilled to witness this milestone for the alpha-mannosidosis community," Mark Stark, treasurer at the International Society for Mannosidosis & Related Disorders (ISMRD), and father of a son living with AM, said in the statement. "I have watched firsthand 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.”
The disease is characterized by the inability of the body's cells to properly break down certain groups of complex sugars, which can affect many of the body's organs and systems. Individuals with alpha-mannosidosis experience impaired hearing, speech, and mobility progressively from childhood to adulthood, though the symptoms of the disease vary significantly from patient to patient.
As a recombinant form of human alpha-mannosidase, velmanase alfa-tycv provides or supplements the patient’s natural alpha-mannosidase. This enzyme is involved in the degradation of mannose–rich oligosaccharides to prevent their accumulation in various tissues in the body.
Chiesi Group received marketing authorization from the European Commission for velmanase alfa-tycv for the treatment of non-neurological manifestations in patients with mild-to-moderate alpha-mannosidosis in 2018.
“(International Society for Mannosidosis & Related Disorders) ISMRD is excited to continue to collaborate with Chiesi,” Stark stated, “And we are thankful for their efforts to develop Lamzede to give patients in the US a much-needed treatment option."