FDA gives green light to Chiesi's Lamzede

FDA gives green light to Chiesi's Lamzede

US regulators have approved Chiesi's Lamzede (velmanase alfa-tycv) as the first enzyme replacement therapy for the treatment of non-central nervous system manifestations of alpha-mannosidosis (AM) in adult and paediatric patients.

The decision follows a thorough priority review process, which began late last year.  

Affecting approximately one in every 500,000 to one in every one million babies born worldwide, AM is a rare inherited metabolic disorder caused by a deficiency of the enzyme alpha-mannosidase. This enzyme is involved in breaking down certain groups of complex sugars. Without enough alpha-mannosidase, these complex sugars accumulate in the body's cells and tissues, leading to progressive damage and dysfunction.

"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," said Giacomo Chiesi, head of Chiesi Global Rare Diseases. "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 effects of AM vary from patient to patient, with common symptoms including recurrent chest and ear infections, hearing loss, distinctive facial features, muscle weakness, skeletal and joint abnormalities, visual abnormalities, and cognitive abnormalities.

Lamzede (velmanase alfa-tycv) is a recombinant form of human alpha-mannosidase designed to replace or support the function of the natural enzyme and prevent the build-up of mannose–rich oligosaccharides in various tissues in the body.

"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 US a much-needed treatment option."

The treatment is already approved for use in Europe where, in 2018, Chiesi received marketing authorisation from the European Commission for Lamzede for the treatment of non-neurological manifestations in patients with mild to moderate AM.

This latest regulatory achievement marks a significant milestone for Chiesi's Boston-based rare disease division, which was formed in 2020 to diversify away from the company's traditional focus on respiratory medicines and further its ambition to grow in the US.