Amicus Therapeutics’ Pompe disease treatment approved for use in UK

Betsy Goodfellow | August 17, 2023 | News story | Medical Communications Amicus Therapeutics, MHRA, NICE, Pompe disease, Rare Diseases 

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 combinatorion 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 U.K.’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.”

Allan Muir, chair of the Board of Trustees of Pompe Support Network, added: “Pompe disease is a rare disease, one that extensively affects all aspects of life for an individual and their family, friends and colleagues. We are very grateful to companies such as Amicus Therapeutics that seek to make meaningful differences for our small community and we welcome new treatments that bring further choice to patients.”

Betsy Goodfellow

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