NHS recommendation to increase Myozyme dose for Pompe infants

NHS recommendation to increase Myozyme dose for Pompe infants

Posted On: 03/05/2022

The NHS has proposed changes to IOPD treatment with Myozyme for Pompe infants.

We have been asked to comment on a recommendation to increase the standard dose of Myozyme for all Pompe children.
This is a fantastic development and is something Allan and the treating physicians have been calling for, for many years. It will bring the UK much closer in line with treatment guidelines in USA, Netherlands, and Taiwan. It will, no doubt, help newly diagnosed children develop and meet their milestones, with much less chance of deterioration during their childhood.


The proposed changes to IOPD treatment with Myozyme are as follows:

  • Newly diagnosed, treatment naïve, IOPD patients.

20mg/kg once weekly alglucosidase alfa (Myozyme)

  • IOPD patients already on enzyme replacement therapy who are not invasively ventilated.

20mg/kg once weekly alglucosidase alfa (Myozyme)

  • IOPD Patients with discernible clinical decline despite treatment with 20mg/kg once weekly. (For example, children with deteriorating respiratory function or worsening muscle function)

40mg/kg once weekly alglucosidase alfa (Myozyme)

There are various stages for the consultation to go through before it is accepted, but we hope that it rapidly leads to acceptance of these changes for our most vulnerable children.