MHRA clears Amicus Therapeutics’ Pompe disease treatment for use in the UK

Amicus Therapeutics has announced that the Medicines and Healthcare products Regulatory Agency (MHRA) in the UK has granted marketing authorisation to Pombiliti (cipaglucosidase alfa) and Opfolda (miglustat) for the treatment of adult patients with late-onset Pompe disease.
Additionally, the National Institute for Health and Care Excellence (NICE) has issued final guidance recommending reimbursement of Pombiliti and Opfolda for use within the NHS in England and Wales.
Prior to its approval, the drug combination was given an Innovation Passport as part of the Innovative Licensing and Access Pathway (ILAP), a Priority Innovative Medicines designation and a positive scientific opinion under the Early Access to Medicines Scheme (EAMS). Bradley Campbell, president and chief executive officer of Amicus Therapeutics, commented: “The MHRA approvals for Pombiliti and Opfolda are a major step forward for adults in the UK living with late-onset Pompe who are seeking new treatments. We are grateful to the global Pompe community who have helped advance this therapy, especially the patients, families and physicians who participated in our clinical studies. The speed in which NICE recommended reimbursement of Pombiliti and Opfolda is reflective of the UK’s Innovative Licensing and Access Pathway, the data behind Pombiliti and Opfolda, the strong collaboration with the reimbursement authorities and the Amicus commitment to bring this therapy to those living with Pompe disease as quickly as possible. I am proud of Amicus’ relentless commitment toward ensuring patient access to our innovative therapies, and we are working as quickly as possible to make Pombiliti and Opfolda commercially available.”
Professor Mark Roberts, consultant neurologist at the Greater Manchester Neurosciences Unit at Salford Royal NHS Foundation Trust, added: “From the positive uptake of the Early Access to Medicines Scheme, we have already seen the impact that this treatment is having for patients. Having widespread access to this treatment is an exciting development for the Pompe community, giving HCPs and patients a new option that exhibits a novel mode of action.”