Pombiliti™ +Opfolda™

Understanding Pombiliti™ + Opfolda™ 

Pombiliti™ (cipaglucosidase alfa-atga) in combination with Opfolda™ (miglustat) is an FDA-approved two-component therapy designed for the treatment of adult patients with late-onset Pompe disease (LOPD) who weigh at least 40 kg and have not shown improvement with their current enzyme replacement therapy (ERT). This therapeutic regimen addresses the underlying enzyme deficiency in Pompe disease, aiming to enhance muscle function and reduce disease progression.

How Pombiliti™ + Opfolda™ Works:

• Enzyme Replacement: Pombiliti™ is a recombinant human acid alpha-glucosidase (rhGAA) enzyme engineered with high levels of bis-Mannose 6-Phosphate (bis-M6P) to facilitate efficient uptake into muscle cells. Once inside the cells, Pombiliti™ is processed into its active form to break down accumulated glycogen, a hallmark of Pompe disease.

• Enzyme Stabilization: Opfolda™ acts as a stabilizer by binding to Pombiliti™ in the bloodstream, reducing its inactivation and ensuring a higher amount of active enzyme reaches the target tissues.

• Administration: The treatment involves intravenous infusion of Pombiliti™ every two weeks, accompanied by oral administration of Opfolda™ prior to each infusion to optimize enzyme stability and activity.

FDA Approval:

• Pombiliti™ in combination with Opfolda™ received FDA approval on September 28, 2023, for the treatment of adult patients with late-onset Pompe disease weighing ≥40 kg who are not improving on their current ERT.

For more information, please visit the Pombiliti™ + Opfolda™ patient website and consult your healthcare provider to determine if this treatment regimen is appropriate for you.