LAMZEDE (velmanase alfa)

Key points

Favourable opinion for maintenance of reimbursement in the enzyme replacement therapy of non-neurological manifestations in patients with mild to moderate alpha-mannosidosis.

The clinical benefit is now substantial, where previously it was moderate.

What therapeutic improvement?

Therapeutic improvement in the management of the disease.

Role in the care pathway?

The treatment objectives of patients with mild to moderate alpha-mannosidosis are to improve their state of health and quality of life by reducing incapacitating symptoms and preventing complications, particularly infectious ones. Symptomatic treatment should be proactive and, in particular, can involve ENT treatment, physiotherapy, orthopaedic surgery, the use of hearing aids and early educational support.

Role of the medicinal product in the care pathway

In patients with a confirmed diagnosis of mild to moderate alpha-mannosidosis, LAMZEDE (velmanase alfa), administered once weekly by intravenous infusion, is a first-line enzyme replacement therapy of non-neurological manifestations of this disease. This replacement therapy, which stabilises progression of the disease, should be implemented at the earliest stages of the disease, hence the importance of the earliest possible diagnosis of this disease.

The optimal treatment duration is currently unknown.

Treatment with LAMZEDE (velmanase alfa) should not be initiated in patients with severe alpha-mannosidosis, which would correspond to off-label use.