FDA approves velmanase alfa-tycv for alpha-mannosidosis

The FDA has approved velmanase alfa-tycv (Lamzede; Chiesi Global Rare Diseases) for use in adult and pediatric patients with alpha-mannosidosis (AM) manifesting in the non-central nervous system.¹

Alpha-mannosidosispresents in approximately 1 in 500,000 to 1 in 1,000,000 babies born worldwide, with symptoms including impairments to speech, hearing, and mobility.In AM, the body is incapable of breaking down specific complex sugar groups, leading to effects on many body organs and symptoms.

Symptoms of AM include recurrent ear and chest infections, distinctive facial features, hearing loss, skeletal and join abnormalities, muscle weakness, and visual and cognitive abnormalities. These symptoms may change as patients age and vary from person to person.

Velmanase alfa-tycvis the first enzyme replacement therapy in the United States granted approval for treating alpha-mannosidosis. It provides natural alpha-mannosidase to the body, which is part of the process of degrading mannose–rich oligosaccharides, keeping them from accumulating in various tissues.

Velmanase alfa-tycvmay cause hypersensitive reactions in some patients, including anaphylaxis. In severe instances, patients should immediately stop taking velmanase alfa-tycvand receive appropriate medical treatment. In mild to moderate reactions to infusion, infusion should be temporarily halted, then slowed, with appropriate medical treatment provided.

Although prior studies had indicated improved clinical outcomes in children taking velmanase alfa-tycv, long-term data was lacking for children aged under 6 years.² The safety and efficacy of the drug in this age group was evaluated in a multicenter, open-label, phase 2 study.

Most adverse events experienced by children taking velmanase alfa-tycvin the trial were mild to moderate, with 4 of the 5 children tested experiencing adverse events. Efficacy assessments showed improved results in at least 1 area for all children with AM. While this data is limited, it indicates long-term safety and efficacy when taking velmanase alfa-tycvfor treating AM.

References

1. Chiesi Global Rare Diseases announces FDA approval of Lamzede® (velmanase alfa-tycv) for alpha-mannosidosis. News release Chiesi. February 16, 2023. Accessed March 2, 2023. https://chiesirarediseases.com/media/chiesi-global-rare-diseases-announces-fda-approval-of-lamzede

2. Guffon N, Konstantopoulou V, Hennermann JB, et al. Long-term safety and efficacy of velmanasealfa treatment in children under 6 years of age with alpha-mannosidosis: a phase 2, open label, multicenter study. J Inherit Metab Dis. 2023. doi:10.1002/jimd.12602