Amicus Therapeutics Gets FDA Approval for Pombiliti + Opfolda

By Chris Wack

Trading in shares of Amicus Therapeutics was halted at $12.88 as the Food and Drug Administration approved Pombiliti + Opfolda 65mg capsules for adults living with late-onset Pompe disease, and who aren't improving on their current enzyme replacement therapy.

The stock hit its 52-week high of $14.10 on Aug. 9, and is up 20% in the past 12 months.

Late-onset Pompe disease is a rare, debilitating, and life-threatening lysosomal disorder caused by a deficiency of the enzyme acid alpha-glucosidase.

Pombiliti is a recombinant human enzyme naturally expressed with high levels of bis-Mannose 6-Phosphate, designed for increased uptake into muscle cells. Opfolda is an enzyme stabilizer designed to stabilize the enzyme in the blood.

The FDA approval was based on clinical data observed from a Phase 3 pivotal study, the only trial in late-onset Pompe disease to study ERT-experienced participants in a controlled setting.

Amicus Therapeutics will launch Pombiliti + Opfolda immediately in the U.S. The FDA previously granted Breakthrough Therapy designation for Pombiliti + Opfolda. It has also been approved for the treatment of adults with late-onset Pompe disease in the E.U. and the U.K.

Write to Chris Wack at chris.wack@wsj.com

(END) Dow Jones Newswires

September 28, 2023 11:16 ET (15:16 GMT)

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