This section highlights FDA-related milestones and regulatory updates for drugs developed by Amicus Therapeutics (FOLD).
Over the past two years, Amicus Therapeutics has reported clinical trial outcomes, regulatory submissions, approvals, and other FDA events for drugs and therapies such as
cipa+mig and Pombiliti. For definitions of regulatory abbreviations such as NDA, BLA, or PDUFA, see the event status legend.
cipa+mig - FDA Regulatory Timeline and Events
cipa+mig is a drug developed by Amicus Therapeutics for the following indication: In adults with late-onset Pompe disease (LOPD) in Muscle and Nerve.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- cipa+mig
- Announced Date:
- June 3, 2025
- Indication:
- In adults with late-onset Pompe disease (LOPD) in Muscle and Nerve.
Announcement
Amicus Therapeutics announced the publication of a post-hoc analysis of data from the ERT-experienced cohort of the PROPEL study of cipaglucosidase alfa-atga + miglustat (cipa+mig) in adults with late-onset Pompe disease (LOPD) in Muscle and Nerve.
AI Summary
Amicus Therapeutics recently announced the publication of a post-hoc analysis of the ERT-experienced group from the PROPEL study in Muscle and Nerve. The analysis focused on adults with late-onset Pompe disease (LOPD) who switched from alglucosidase alfa to cipaglucosidase alfa-atga plus miglustat (cipa+mig). Results showed that patients who switched experienced improvements or stability in many measures, including the 6-minute walk distance, muscle strength tests, and fatigue scores, along with positive changes in biomarker levels. In contrast, those who continued treatment with alglucosidase alfa generally showed worsening or only stable outcomes in several areas, especially lung function. These findings are important because LOPD is a progressive disease, and the study suggests that switching to cipa+mig may better support patient performance and quality of life over time.
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Pombiliti - FDA Regulatory Timeline and Events
Pombiliti is a drug developed by Amicus Therapeutics for the following indication: For Pompe disease.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- Pombiliti
- Announced Date:
- June 25, 2025
- Indication:
- For Pompe disease
Announcement
Amicus Therapeutics announced that Japan's Ministry of Health, Labour and Welfare (MHLW) has approved Pombiliti (cipaglucosidase alfa) + Opfolda (miglustat) for the treatment of adult patients with late-onset Pompe disease (LOPD).
AI Summary
Amicus Therapeutics recently announced that Japan’s Ministry of Health, Labour and Welfare (MHLW) has approved the use of Pombiliti (cipaglucosidase alfa) combined with Opfolda (miglustat) for treating adult patients with late-onset Pompe disease (LOPD). This two-part therapy offers a new treatment option by using Pombiliti, a specially designed recombinant enzyme that enhances uptake into muscle cells, along with Opfolda, which acts as an enzyme stabilizer to prevent loss of activity in the blood. The approval, based on strong clinical data from the Phase 3 PROPEL study that included both previously treated and untreated patients, marks a significant development for the Pompe community in Japan. This new therapeutic option is intended to improve the quality of life for adults with LOPD, providing hope to patients, their families, and healthcare providers alike.
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