alglucosidase alfa (Myozyme®)

Following a full submission

alglucosidase alfa (Myozyme) is not recommended for use within NHS Scotland for the treatment of Pompe disease (acid a-glucosidase deficiency).

Treatment in patients with the infantile-form of Pompe disease significantly improved survival compared with historical controls. The evidence is less clear for patients who are already receiving ventilatory support or who have the late-onset form of the disease. The economic case has not been demonstrated.

The SMC orphan drug policy requires manufacturers to make complete submissions to allow a comprehensive product assessment similar to all other drug submissions. However, in addition to the usual assessment of clinical and cost-effectiveness, SMC may consider additional factors specific to orphan products. Within this context the particular features of the condition and population receiving the technology and whether a drug can reverse (rather than stabilise) the condition or bridge a gap to a definitive therapy may also be considered.

SMC considered the submission in the context of its orphan drug policy.