PSN have had meetings with representatives of Sanofi, Amicus/BioMarin, the Department of Health and Social Care and the NHS. The agreed position of NHS and DHSC is that all paediatric patients and adults with infantile-onset Pompe disease (IOPD) will be prioritised for drug allocation.
All adult patients with late-onset Pompe disease (LOPD) will receive infusions at their usual dose every four weeks (half the usual frequency) while the shortage continues, rather than the current regimen of infusions every two weeks. Clinical trial patients will not be prioritised.
PSN have raised the issue of adults whose condition is more precarious and asked whether they can be given any priority, because missing infusions could potentially cause significant irreversible deterioration.
Sanofi has confirmed that a drug delivery is expected in mid-September. However, this delivery will not meet the overall demand, and the exact quantity to be delivered has yet to be confirmed.
The drug supply issue has been escalated to ministerial level in the UK.
Additionally, there may be capacity for a limited number of patients to switch to the Amicus/BioMarin ERT, subject to patient eligibility and drug availability. This would have to be carefully managed so as not to overstretch Amicus/BioMarin's own supply chain.
PSN have requested regular updates from Sanofi, the NHS Supply Team and the lead commissioner and will post additional information as it becomes available.
Please contact your specialist team if you have any individual concerns.