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MHRA and NICE approve two-component therapy for late-onset Pompe disease

17th August 2023

A treatment combining cipaglucosidase alfa (Pombiliti) and miglustat (Opfolda) for adults living with late-onset Pompe disease has been granted marketing authorisation by the UK’s Medicines and Healthcare products Regulatory Agency (MHRA).

Cipaglucosidase alfa is a long-term enzyme replacement therapy (ERT) used in combination with the enzyme stabiliser miglustat for adults with late-onset Pompe disease. This rare, inherited lysosomal disorder is caused by deficiency of the enzyme acid alpha-glucosidase (GAA).

The approval comes as the National Institute for Health and Care Excellence (NICE) issued final guidance recommending reimbursement of Pombiliti + Opfolda for use within the NHS in England and Wales.

NICE concluded that the cost-effectiveness estimates for Pombiliti + Opfolda showed a positive net health benefit and recommended Pombiliti + Opfolda for adults with late-onset Pompe disease as first line and later lines of therapy.

The MHRA approval and NICE decisions were based on clinical data from the Phase 3 pivotal PROPEL study in which Pombiliti + Opfolda was associated with demonstrable improvements in both musculoskeletal and respiratory measures.

The time to market for Pombiliti + Opfolda was accelerated after it was granted an Innovation Passport under the Innovative Licensing and Access Pathway, a Priority Innovative Medicines designation and a positive scientific opinion under the Early Access to Medicines Scheme. This enabled healthcare professionals to prescribe the treatment prior to marketing authorisation based on clinical factors for patients with a clear unmet need.

In Pompe disease, reduced or absent levels of GAA lead to the accumulation of the substrate glycogen in the lysosomes of muscles and other tissues, leading to skeletal muscle weakness and progressive respiratory involvement.

Professor Mark Roberts, consultant neurologist at the Greater Manchester Neurosciences Unit at Salford Royal NHS Foundation Trust, commented: ‘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.’

Referring to this ‘major step forward’ for late-onset Pompe disease treatment, Bradley Campbell, president and chief executive officer of Amicus Therapeutics, said: ‘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. 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.’

Pombiliti + Opfolda will both be added to the Orphan Register held by the MHRA and will benefit from 10 years of market exclusivity.

The European Commission granted approval for Opfolda for adults with late-onset Pompe disease in late June, following its approval of Pombiliti in March 2023.