Amicus announces approval, launch of new Pompe disease therapy in U.K.

Amicus Therapeutics announced that the Medicines and Healthcare products Regulatory Agency, MHRA, of the United Kingdom has granted marketing authorizations for Pombiliti + Opfolda for adults living with late-onset Pompe disease. The indication for Pombiliti is a long-term enzyme replacement therapy, ERT, used in combination with miglustat for adults with late-onset Pompe disease, LOPD. The indication for Opfolda is an enzyme stabilizer of cipaglucosidase alfa long-term enzyme replacement therapy for adults with LOPD. In addition, the National Institute for Health and Care Excellence, NICE issued final guidance recommending reimbursement of Pombiliti + Opfolda for use within the National Health Service, NHS in England and Wales. As stated in the guidance, NICE concluded that the cost-effectiveness estimates for Pombiliti + Opfolda showed a positive net health benefit and recommended Pombiliti + Opfolda for adults with LOPD as first line and later lines of therapy. “The MHRA approvals for Pombiliti and Opfolda are a major step forward for adults in the U.K. 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,” stated Bradley Campbell, President and Chief Executive Officer of Amicus Therapeutics, Inc. “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.”