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Amicus Therapeutics Developed Breakthrough Pombiliti™ Treatment for Pompe Disease

Amicus Therapeutics unveiled Pombiliti™, a groundbreaking combination of Cipaglucosidase alfa-ATGA and Opfolda™ 65mg capsules, designed to assist adults dealing with late-onset Pompe disease (LOPD).

This dual therapy is specifically designed for adults diagnosed with late-onset Pompe disease (LOPD) weighing ≥40 kg, particularly those who have not experienced improvement while on their current enzyme replacement therapy (ERT).

Pombiliti + Opfolda represents a distinctive two-part therapy. Pombiliti, a recombinant human GAA enzyme (rhGAA), boasts naturally high levels of bis-M6P (Mannose 6-Phosphate), optimising its absorption into muscle cells. 

Once inside, Pombiliti undergoes proper processing into its most potent and mature form, effectively breaking down glycogen. Meanwhile, Opfolda serves as an enzyme stabiliser, specifically engineered to enhance the enzyme's stability within the bloodstream.

Late-onset Pompe disease, a rare and life-threatening lysosomal disorder, emerges due to a deficiency in the enzyme acid alpha-glucosidase (GAA). When GAA levels drop, glycogen accumulates within muscle cell lysosomes, leading to extensive muscle damage. 

The severity of the disease spans a spectrum, marked primarily by manifestations like skeletal muscle weakness and progressive respiratory complications. This condition can be debilitating, causing a gradual decline in muscle strength, particularly affecting skeletal muscles and those crucial for breathing.

The US FDA has approved Pombiliti™ (cipaglucosidase alfa-atga) + Opfolda™ (miglustat),  marking the first and sole two-component therapy available for eligible adults coping with late-onset Pompe disease.  

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