Amicus Therapeutics : FDA Approves Pombiliti + Opfolda 65mg Capsules For Late-onset Pompe Disease

(RTTNews) - Amicus Therapeutics (FOLD) said that the U.S. Food and Drug Administration has approved Pombiliti (cipaglucosidase alfa-atga) + Opfolda (miglustat) 65mg capsules. The two-component therapy is indicated for adults living with late-onset Pompe disease or LOPD weighing greater than or equal to 40 kg and who are not improving on their current enzyme replacement therapy or ERT.

Late-onset Pompe disease is a rare, debilitating, and life-threatening lysosomal disorder caused by a deficiency of the enzyme acid alpha-glucosidase (GAA). Reduced levels of GAA lead to the accumulation of the substrate glycogen in the lysosomes of muscle cells and glycogen buildup causes muscle damage.

Amicus Therapeutics said it will launch Pombiliti + Opfolda immediately in the U.S. The FDA previously granted Breakthrough Therapy designation for Pombiliti + Opfolda. Pombiliti + Opfolda has also been approved for the treatment of adults with LOPD in the European Union and the United Kingdom.

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