Pharma Focus Asia

WuXi Biologics Congratulates Amicus Therapeutics on FDA Approval for Pompe Disease Treatment

Tuesday, October 03, 2023

WuXi Biologics applauds Amicus Therapeutics on FDA approval for innovative Pombiliti™ + Opfolda™ Therapy for late-onset pompe disease.

This innovative two-component therapy is specifically intended for adults with late-onset Pompe disease (LOPD) weighing ≥40 kg who are not experiencing improvements with their current enzyme replacement therapy (ERT).

The strong partnership between the WuXi Biologics team and Amicus has bought this new treatment to market. The joint efforts have brought this medicine from an idaea, to a molecule, to clinical manufacturing and commercial supply, fulfilling the commitment to late-onset Pompe disease patients.

Pombiliti™ + Opfolda™ combines cipaglucosidase alfa-atga, a bis-M6P-enriched rhGAA that enhances high-affinity uptake through the M6P receptor while maintaining its capacity for processing into the enzyme's most active form, with miglustat, an oral enzyme stabiliser engineered to minimise enzyme activity loss in the bloodstream.

Pompe disease, an inherited lysosomal disorder stemming from a deficiency of the enzyme acid alpha-glucosidase (GAA), leads to the accumulation of glycogen in cells, contributing to the disease's clinical manifestations. Pompe disease ranges from a rapidly deteriorating infantile form with significant heart function impact to a more gradually progressing late-onset variant primarily affecting skeletal muscles and gradually involving the respiratory system.

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