MYOZYME 50 mg (alglucosidase alfa)

Key points

Favourable opinion for reimbursement in the late-onset form of Pompe disease.

Long-term enzyme replacement therapy (ERT) in patients with a confirmed diagnosis of Pompe disease (acid α-glucosidase deficiency).

Myozyme is indicated in adults and paediatric patients of all ages.

The re-evaluation only concerns late-onset forms of Pompe disease.

Role in the care pathway?

The multidisciplinary management of the disease is coordinated by a hospital physician in liaison with a reference or expert centre for metabolic or neuromuscular diseases and in liaison with the patient’s primary care physician.

The specific treatment of Pompe disease is based on enzyme replacement therapy.

The objectives of treatment are as follows: to improve or stabilise hypertrophy and cardiac function in the event of initial damage (infantile-onset form); to stabilise or slow worsening of muscle weakness and avoid or delay loss of walking ability; to stabilise or slow deterioration of respiratory function and avoid or delay the need for ventilatory support.

Role of the medicinal product in the care pathway

MYOZYME (alpha alglucosidase) is the only enzyme replacement therapy with an MA in Pompe disease.