On Wednesday 26th April Amicus Therapeutics announced the EU Committee for Medicinal Products for Human Use (CHMP) have recommended approval of Opfolda for Pompe disease, that is the Miglustat pill that accompanies their latest ERT for Pompe disease (Pombiliti). The European Commission is therefore expected to give marketing authorisation for the drug combination in the summer and we will expect the UK regulators (NICE) to follow suit. NICE have a committee meeting to appraise Pombiliti plus Opfolda on 3rd May 2023, so it is likely that approval will be given at the same time as the EU.
Many in the Pompe community are already experiencing this drug combination since it was approved under an Early Access to Medicines Scheme (EAMS), so the main difference to the Pompe community will be that homecare should again allow independence from the nursing service, either by self-infusion, or by the nurse leaving after canulating for the infusion. Also, the NHS will no longer receive the combination therapy for free. We are very keen to hear your views and experiences of the second-generation therapies that have been rolled out over the last year from Sanofi and Amicus.
Please do contact us if you have any experiences, good or bad, that you would like to share: firstname.lastname@example.org