Amicus Therapeutics Receives FDA Approval for Pombiliti + Opfolda in Late-Onset Pompe Disease

by Warren Seah

By Chris Wack

Shares of Amicus Therapeutics have been temporarily halted at $12.88 following the Food and Drug Administration’s (FDA) approval of Pombiliti + Opfolda 65mg capsules for adults living with late-onset Pompe disease. This approval is especially significant for patients who are not seeing improvement with their current enzyme replacement therapy.

Amicus Therapeutics has seen substantial growth in its stock, with a 20% increase over the past year and reaching a 52-week high of $14.10 on August 9th.

Late-onset Pompe disease is a rare and severe lysosomal disorder that poses significant threats to patients’ lives. It is caused by a deficiency of the enzyme acid alpha-glucosidase.

Pombiliti offers a recombinant human enzyme naturally expressed with high levels of bis-Mannose 6-Phosphate. This design allows for enhanced muscle cell uptake. Additionally, Opfolda acts as an enzyme stabilizer, ensuring the enzyme remains stable in the blood.

The FDA’s approval was influenced by promising clinical data acquired from a Phase 3 pivotal study. This trial exclusively studied participants with late-onset Pompe disease who had prior experience with enzyme replacement therapy in a controlled setting.

Amicus Therapeutics wasted no time and will promptly launch Pombiliti + Opfolda in the United States. The FDA has previously granted Breakthrough Therapy designation for this treatment combination. Moreover, it has been approved for managing late-onset Pompe disease in adults within both the European Union and the United Kingdom.

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