Amicus Therapeutics Presents New Long-term Data for both Galafold® (migalastat) and POMBILITI® (cipaglucosidase alfa-atga) + OPFOLDA® (miglustat) at the 22nd Annual WORLDSymposium™ 2026

Rhea-AI Summary

Amicus Therapeutics (Nasdaq: FOLD) presented new long-term clinical and real-world data for Galafold (migalastat) in Fabry disease and POMBILITI + OPFOLDA (cipaglucosidase alfa plus miglustat) in late-onset Pompe disease at the 22nd Annual WORLDSymposium™ 2026. Highlights include registry analyses and long-term outcomes including 208-week and 90-month data across oral presentations and poster sessions. Galafold is approved in >40 countries and is indicated for adults with amenable GLA variants; Amicus estimates ~35–50% of patients have amenable variants. Safety and usage considerations for both therapies are included.

Positive

• Presentation of 208-week outcomes for cipaglucosidase alfa plus miglustat
• Publication of 90-month muscle and pulmonary data in Pompe disease
• Real-world analyses from the global followME Pathfinders registry for migalastat
• Galafold approved in more than 40 countries
• Company estimates 35–50% of Fabry patients have amenable GLA variants

Negative

• POMBILITI carries a boxed warning for cardiopulmonary risk during infusion
• POMBILITI + Opfolda is contraindicated in pregnancy
• Galafold is not recommended for severe renal impairment or dialysis patients
• Galafold lacks sufficient clinical data for use in pregnancy and pediatric patients
• Common adverse reactions include headache, nausea, and pyrexia for both therapies

News Market Reaction

-0.07%

1 alert

-0.07% News Effect

On the day this news was published, FOLD declined 0.07%, reflecting a mild negative market reaction.

Data tracked by StockTitan Argus on the day of publication.

Key Figures

Capsule strength: 123 mg Fabry amenable variants: 35–50% Follow-up duration: 208 weeks +5 more

8 metrics

Capsule strength 123 mg Galafold (migalastat) oral capsules

Fabry amenable variants 35–50% Estimated share of Fabry patients with amenable GLA variants

Follow-up duration 208 weeks Outcomes for cipaglucosidase alfa plus miglustat from PROPEL baseline

Long-term follow-up 90 months Pulmonary and muscle outcomes in ATB200-02 Pompe study

Body weight threshold ≥40 kg Eligibility for Pombiliti + Opfolda in late-onset Pompe

Common adverse reactions ≥10% Most frequent side effects reported with Galafold treatment

Adverse reactions threshold ≥5% Most common Pombiliti adverse reactions (e.g., headache, diarrhea, fatigue)

Conference edition 22nd Annual WORLDSymposium where data are presented

Market Reality Check

Price: $14.27 Vol: Volume 7,773,807 vs 20-da...

normal vol

$14.27 Last Close

Volume Volume 7,773,807 vs 20-day average 9,792,434, indicating below-normal activity ahead of this update. normal

Technical Trading above longer-term trend: price 14.29 vs 200-day MA 8.53, also within 1% of the 52-week high at 14.38.

Peers on Argus

FOLD was flat at 0% change while several biotech peers showed gains (e.g., TVTX ...

FOLD was flat at 0% change while several biotech peers showed gains (e.g., TVTX +6.45%, CGON +5.17%, AGIO +3.61%). This suggests today’s setup is more stock-specific, with FOLD already trading near its 52-week high and pending an all-cash buyout at $14.50 per share from prior news.

Historical Context

5 past events · Latest: Dec 19 (Positive)

Pattern 5 events

Date	Event	Sentiment	Move	Catalyst
Dec 19	Acquisition announcement	Positive	+30.2%	BioMarin all-cash acquisition at $14.50 per share valued at $4.8B.
Nov 04	Earnings results	Positive	+1.5%	Q3 2025 revenue growth, GAAP profitability, and reaffirmed full-year guidance.
Oct 31	Investor conferences	Neutral	+1.5%	Management participation in November healthcare investor conferences.
Oct 23	Earnings notice	Neutral	-0.6%	Announcement of upcoming Q3 2025 earnings call and webcast logistics.
Sep 08	Clinical data update	Positive	-0.1%	Four-year PROPEL extension data showing durable efficacy in late-onset Pompe.

Pattern Detected

News tied to financial performance and the BioMarin acquisition generally aligned with positive price moves, while detailed clinical data previously saw little reaction.

Recent Company History

Over the last six months, FOLD’s trajectory has been shaped by commercial execution and its pending acquisition. On Dec 19, 2025, BioMarin agreed to acquire Amicus for $14.50 per share, a deal that triggered a +30.21% move and highlighted the value of Galafold and Pombiliti + Opfolda. Strong Q3 2025 results and guidance on Nov 4 also drew a modest positive reaction. By contrast, earlier long-term clinical data for Pombiliti + Opfolda on Sep 8 had almost no price impact.

Market Pulse Summary

This announcement highlights extensive long-term and real-world data for Galafold and Pombiliti + Op...

Analysis

This announcement highlights extensive long-term and real-world data for Galafold and Pombiliti + Opfolda, including 208-week and 90-month outcomes and broader registry work in Fabry and Pompe disease. In the context of a pending all-cash acquisition at $14.50 per share and prior strong revenue growth, these updates primarily deepen the evidence base for approved therapies. Investors may focus on durability, safety, and real-world effectiveness metrics as key markers of the franchises’ long-term profile within the combined company.

Key Terms

pharmacokinetic, pharmacodynamics, post-marketing surveillance, enzyme replacement therapy, +4 more

8 terms

pharmacokinetic medical

"to evaluate the safety, pharmacokinetics, pharmacodynamics and efficacy of migalastat"

Pharmacokinetic describes how a drug moves through and leaves the body — how it is absorbed, spread to tissues, broken down and excreted — like tracking a package from pickup to delivery and disposal. For investors, these properties determine effective dose, safety risks, how often a medicine must be taken, and how reliably it works, which in turn influence clinical trial success, regulatory approval chances, production complexity and a drug’s commercial value.

pharmacodynamics medical

"to evaluate the safety, pharmacokinetics, pharmacodynamics and efficacy of migalastat"

Pharmacodynamics is how a drug actually affects the body — the strength, type and duration of its effects and the relationship between dose and response. Think of it like how turning a thermostat changes room temperature: it shows what the drug does and how much is needed to get the desired effect. Investors care because these properties drive clinical success, dosing convenience, safety profile and competitive advantage, all of which influence commercial potential and regulatory approval.

post-marketing surveillance regulatory

"Long-term safety and effectiveness of migalastat in patients with Fabry disease: results from the Korean post-marketing surveillance"

Post-marketing surveillance is the ongoing monitoring of a drug, medical device, or vaccine after it has been approved and sold to the public to track real-world safety, side effects, and long-term performance. It matters to investors because new safety findings can trigger label changes, recalls, or additional costs and affect sales and stock value—think of it as continued quality control and feedback from customers that can change a product’s market prospects.

enzyme replacement therapy medical

"versus intravenous enzyme replacement therapies (ERTs) for clinical measures in Fabry disease"

Enzyme replacement therapy is a medical treatment that involves providing patients with artificial versions of natural enzymes their bodies are missing or not producing enough of. This approach can help manage certain health conditions by restoring essential functions, similar to replacing a faulty part in a machine to keep it running smoothly. For investors, advancements or approvals in this therapy can signal progress in biotech innovation and potential market growth.

open-label medical

"ATB200-02, an open-label phase I/II study"

Open-label describes a situation where everyone involved in a study or process knows the full details, such as who is receiving a treatment or intervention. For investors, understanding whether a project or product is open-label helps gauge the level of transparency and potential biases, influencing trust and decision-making. It’s like knowing whether a test or experiment is conducted openly or behind closed doors.

phase I/II medical

"ATB200-02, an open-label phase I/II study"

"Phase I/II" describes early stages of testing a new medicine or treatment, where researchers first evaluate its safety and then begin to see if it works. For investors, these phases are important because they indicate whether a product is progressing toward potential approval and commercialization, which can impact future value and success prospects. These stages help gauge how close a new treatment is to reaching the market.

accelerated approval regulatory

"This indication is approved under accelerated approval based on reduction in kidney"

Accelerated approval is a process that allows new medical treatments to be approved more quickly than usual if they address serious or life-threatening conditions and show promising early results. For investors, it signals that a treatment may reach the market sooner, potentially boosting a company's prospects, but it also involves some uncertainty since full evidence of effectiveness is still being gathered.

globotriaosylceramide medical

"based on reduction in kidney interstitial capillary cell globotriaosylceramide (KIC GL-3) substrate"

Globotriaosylceramide is a specific fat molecule that builds up inside cells when a particular enzyme is missing or not working, causing a genetic disorder. For investors, it matters because the amount of this molecule acts like a thermometer for disease activity and treatment success—drugs that lower its level can drive clinical benefits, regulatory approvals, and market value. Think of it as a visible leak that shows whether a medical “plumbing” fix is working.

AI-generated analysis. Not financial advice.

PRINCETON, N.J., Feb. 03, 2026 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq: FOLD), today announced the presentation of new data from clinical and real-world studies of Galafold^® (migalastat) in Fabry disease and POMBILITI^® + OPFOLDA^® (cipaglucosidase alfa plus miglustat) in late-onset Pompe disease. Data are being presented at the 22nd Annual WORLDSymposium™ 2026.

“Amicus continues to advance the science and understanding of both Fabry and Pompe diseases, and we are proud to showcase new data for our medicines at this year’s WORLDSymposium. These new data add to the growing body of evidence supporting the important role of Galafold and Pombiliti + Opfolda,” said Jeff Castelli, PhD, Chief Development Officer, Amicus Therapeutics, Inc. “We are immensely grateful to the patients, their families, and investigators whose partnership makes scientific research and advances possible.”

Oral Presentations:

Abstract Title	Disease	Presenter	Date and Time
Real-world effectiveness of migalastat versus enzyme replacement therapy in previously treatment-naïve patients with Fabry disease: analyses of matched populations from the global followME Pathfinders registry	Fabry	Peter Nordbeck, University of Wuerzburg, Wuerzburg, Germany	Thursday, February 5, 8:30 a.m. PST
208-week outcomes of cipaglucosidase alfa plus miglustat in patients with late-onset Pompe disease treated from PROPEL baseline: muscle function and biomarkers	Pompe	Tahseen Mozaffar, University of California, Irvine, Irvine, CA, USA	Friday, February 6, 1:20 p.m. PST

Poster Sessions

Fabry Disease:

Abstract Title	Presenter	Date and Time
Exploring the lived experience of the Fabry community in Czechia (Poster #378)	Christopher Wingrove, Amicus Therapeutics, Inc., Marlow, UK	Tuesday, February 3, 3:30 – 5:30 p.m. PST
followME Fabry Pathfinders registry: cardiac and renal effectiveness in a multi-national, multi-center cohort of patients on migalastat treatment for 5 years (Poster #266)	Peter Nordbeck, University of Wuerzburg, Wuerzburg, Germany	Thursday, February 5, 3:30 – 5:30 p.m. PST
Real-world effectiveness of migalastat versus enzyme replacement therapy in previously treatment-naïve patients with Fabry disease: analyses of matched populations from the global followME Pathfinders registry (Poster #267)	Peter Nordbeck, University of Wuerzburg, Wuerzburg, Germany	Thursday, February 5, 3:30 – 5:30 p.m. PST
Adjusted migalastat dose regimens in patients with Fabry disease and amenable GLA variants with severe renal impairment, or with end-stage renal disease and receiving hemodialysis/hemodiafiltration (HD/HDF): pharmacokinetic (PK) and safety results from a protocol-specified interim analysis of the RENEW study (Poster #187)	Franklin K. Johnson, Amicus Therapeutics, Inc., Princeton, NJ, USA	Thursday, February 5, 3:30 – 5:30 p.m. PST
Trial in progress: an open-label study (AT1001-033; ASPIRE II) to evaluate the safety, pharmacokinetics, pharmacodynamics and efficacy of migalastat in pediatric patients with Fabry disease (Poster #384)	Haichen Yang, Amicus Therapeutics, Inc., Philadelphia, PA, USA	Thursday, February 5, 3:30 – 5:30 p.m. PST
A structured methodology for evaluating patient-reported outcomes across a treatment program: a comparative application in migalastat (Poster #127)	Vera Gielen, Amicus Therapeutics, Inc., Marlow, UK	Thursday, February 5, 3:30 – 5:30 p.m. PST
Clinical characterization and healthcare usage in Fabry disease using Swedish national registers (Poster #118)	Emma Flordal Thelander, Amicus Therapeutics, Inc., Stockholm, Sweden	Thursday, February 5, 3:30 – 5:30 p.m. PST
followME Fabry Pathfinders real-world registry in Spain and Portugal: cardiac and renal outcomes with migalastat in patients with Fabry disease (Poster #22)	Olga Azevedo, Hospital Senhora da Oliveira, Guimarães, Portugal	Thursday, February 5, 3:30 – 5:30 p.m. PST
Matching-adjusted indirect comparisons (MAICs) and network meta-analyses (NMAs) of the oral small-molecule chaperone migalastat versus intravenous enzyme replacement therapies (ERTs) for clinical measures in Fabry disease (Poster #128)	Vera Gielen, Amicus Therapeutics, Inc., Marlow, UK	Thursday, February 5, 3:30 – 5:30 p.m. PST
Long-term safety and effectiveness of migalastat in patients with Fabry disease: results from the Korean post-marketing surveillance (Poster #168)	Geu-Ru Hong, Severance Cardiovascular Hospital, Seoul, Korea	Thursday, February 5, 3:30 – 5:30 p.m. PST

Pompe Disease:

Abstract Title	Presenter	Date and Time
208-week outcomes of cipaglucosidase alfa plus miglustat in patients with late-onset Pompe disease treated from PROPEL baseline: pulmonary function (Poster #205)	Priya S. Kishnani, Duke University, Durham, NC, USA	Thursday, February 5, 3:30 – 5:30 p.m. PST
208-week outcomes of cipaglucosidase alfa plus miglustat in patients with late-onset Pompe disease treated from PROPEL baseline: Muscle function and biomarkers (Poster #257)	Tahseen Mozaffar, University of California, Irvine, Irvine, CA, USA	Thursday, February 5, 3:30 – 5:30 p.m. PST
90-month muscle function and biomarker outcomes with cipaglucosidase alfa plus miglustat (cipa+mig) in adults with Pompe disease in ATB200-02, an open-label phase I/II study (Poster #313)	Benedikt Schoser, Friedrich-Baur-Institute, Ludwig-Maximilians-University, Munich, Germany	Thursday, February 5, 3:30 – 5:30 p.m. PST
90-month pulmonary function outcomes with cipaglucosidase alfa plus miglustat (cipa+mig) in adults with Pompe disease in ATB200-02, an open-label phase I/II study (Poster #206)	Priya S. Kishnani, Duke University, Durham, NC, USA	Thursday, February 5, 3:30 – 5:30 p.m. PST
90-month physician and patient-reported outcomes with cipaglucosidase alfa plus miglustat (cipa+mig) in adults with Pompe disease in ATB200-02, an open-label phase I/II study (Poster #299)	Mark Roberts, Salford Royal NHS Foundation Trust, Salford, UK	Thursday, February 5, 3:30 – 5:30 p.m. PST
From frustration to function: reimagining registry value with individual monitoring dashboards to visualize disease progression in Pompe disease (Poster #247)	Paul McIntosh, University of Pennsylvania, Philadelphia, PA, USA	Thursday, February 5, 3:30 – 5:30 p.m. PST
ROSSELLA: an ongoing open-label, multicenter, global trial to study next-generation treatment of infantile-onset Pompe disease combining enzyme replacement with a stabilizing iminosugar (Poster #244)	Thorsten Marquardt, University of Münster, Münster, Germany	Thursday, February 5, 3:30 – 5:30 p.m. PST
Impact of mobility-aid use on late-onset Pompe disease (LOPD) patient experience: insights from patient interviews (Poster #178)	Derralynn Hughes, Royal Free London NHS Foundation Trust, University College London, London, UK	Thursday, February 5, 3:30 – 5:30 p.m. PST
Delayed diagnosis and missed opportunities: results from an analysis of the diagnostic journey for LOPD in the UK (Poster #82)	Patrick Deegan, Cambridge University Hospitals NHS Foundation Trust (CUH), Cambridge, UK	Thursday, February 5, 3:30 – 5:30 p.m. PST

About Galafold
Galafold^® (migalastat) 123 mg capsules is an oral pharmacological chaperone of alpha-Galactosidase A (alpha-Gal A) for the treatment of Fabry disease in adults who have amenable galactosidase alpha gene (GLA) variants. In these patients, Galafold works by stabilizing the body’s own dysfunctional enzyme so that it can clear the accumulation of disease substrate. Globally, Amicus Therapeutics estimates that approximately 35 to 50 percent of people living with Fabry disease may have amenable GLA variants, though amenability rates within this range vary by geography. Galafold is approved in more than 40 countries around the world, including the U.S., EU, U.K., and Japan.

U.S. INDICATIONS AND USAGE
Galafold is indicated for the treatment of adults with a confirmed diagnosis of Fabry disease and an amenable galactosidase alpha gene (GLA) variant based on in vitro assay data.

This indication is approved under accelerated approval based on reduction in kidney interstitial capillary cell globotriaosylceramide (KIC GL-3) substrate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.

U.S. IMPORTANT SAFETY INFORMATION

ADVERSE REACTIONS: The most common adverse reactions reported with Galafold (≥10%) were headache, nasopharyngitis, urinary tract infection, nausea and pyrexia. 

USE IN SPECIFIC POPULATIONS: There is insufficient clinical data on Galafold use in pregnant women to inform a drug-associated risk for major birth defects and miscarriage. Advise women of the potential risk to a fetus. It is not known if Galafold is present in human milk. Therefore, the developmental and health benefits of breastfeeding should be considered along with the mother’s clinical need for Galafold and any potential adverse effects on the breastfed child from Galafold or from the underlying maternal condition. Galafold is not recommended for use in patients with severe renal impairment or end-stage renal disease requiring dialysis. The safety and effectiveness of Galafold have not been established in pediatric patients. To report Suspected Adverse Reactions, contact Amicus Therapeutics at 1-877-4AMICUS or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. For additional information about Galafold, please see the full U.S. Prescribing Information.

About Pombiliti + Opfolda
Pombiliti + Opfolda, is a two-component therapy that consists of cipaglucosidase alfa-atga, a bis-M6P-enriched rhGAA that facilitates high-affinity uptake through the M6P receptor while retaining its capacity for processing into the most active form of the enzyme, and the oral enzyme stabilizer, miglustat, that’s designed to reduce loss of enzyme activity in the blood.

U.S. INDICATIONS AND USAGE
POMBILITI in combination with OPFOLDA is indicated for the treatment of adult patients with late-onset Pompe disease (lysosomal acid alpha-glucosidase [GAA] deficiency) weighing ≥40 kg and who are not improving on their current enzyme replacement therapy (ERT).

SAFETY INFORMATION

HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS: Appropriate medical support measures, including cardiopulmonary resuscitation equipment, should be readily available. If a severe hypersensitivity reaction occurs, POMBILITI should be discontinued immediately and appropriate medical treatment should be initiated. INFUSION-ASSOCIATED REACTIONS (IARs): If severe IARs occur, immediately discontinue POMBILITI and initiate appropriate medical treatment. RISK OF ACUTE CARDIORESPIRATORY FAILURE IN SUSCEPTIBLE PATIENTS: Patients susceptible to fluid volume overload, or those with acute underlying respiratory illness or compromised cardiac or respiratory function, may be at risk of serious exacerbation of their cardiac or respiratory status during POMBILITI infusion. See PI for complete Boxed Warning. CONTRAINDICATION: POMBILITI in combination with Opfolda is contraindicated in pregnancy. EMBRYO-FETAL TOXICITY: May cause embryo-fetal harm. Advise females of reproductive potential of the potential risk to a fetus and to use effective contraception during treatment and for at least 60 days after the last dose. Adverse Reactions: Most common adverse reactions ≥ 5% are headache, diarrhea, fatigue, nausea, abdominal pain, and pyrexia. Please see full PRESCRIBING INFORMATION, including BOXED WARNING, for POMBILITI (cipaglucosidase alfa-atga) and full PRESCRIBING INFORMATION for OPFOLDA (miglustat).

About WORLDSymposium
WORLDSymposium is designed for basic, translational and clinical researchers, patient advocacy groups, clinicians, and all others who are interested in learning more about the latest discoveries related to lysosomal diseases and the clinical investigation of these advances. Each year, WORLDSymposium presents the latest information from basic science, translational research, and clinical trials for lysosomal diseases. For more information, please visit www.worldsymposia.org.

About Amicus Therapeutics
Amicus Therapeutics (Nasdaq: FOLD) is a leading, global biotechnology company with a clear and compelling mission: to develop and deliver transformative medicines for people living with rare diseases. With extraordinary patient focus, Amicus strives to redefine expectations in rare disease. For more information please visit the company’s website at www.amicusrx.com, and follow on LinkedIn.

CONTACT:

Investors:
Amicus Therapeutics
Andrew Faughnan
Vice President, Investor Relations
afaughnan@amicusrx.com
(609) 662-3809

Media:
Amicus Therapeutics
Brendan McEvoy
Executive Director, External Communications
bmcevoy@amicusrx.com
(609) 662-5005

CRPA-PF-US-26-00001-1

FOLD-G

FAQ

What long-term Pompe disease data did Amicus (FOLD) present at WORLDSymposium 2026?

Amicus presented 208-week outcomes and 90-month follow-up data for cipaglucosidase alfa plus miglustat. According to Amicus, the data cover muscle function, pulmonary function, biomarkers, and patient-reported outcomes across PROPEL and ATB200-02 cohorts.

What Fabry disease evidence for Galafold did Amicus (FOLD) show at the conference?

Amicus presented real-world effectiveness analyses from the global followME Pathfinders registry comparing migalastat to enzyme replacement therapy. According to Amicus, presentations include cardiac and renal outcomes and matched-population analyses for treatment-naïve patients.

Is Galafold approved and which patients are eligible according to Amicus (FOLD)?

Galafold is approved in more than 40 countries for adults with amenable GLA variants. According to Amicus, approximately 35–50% of people with Fabry disease may have amenable variants, subject to geographic variation.

What key safety warnings apply to POMBILITI + OPFOLDA as noted by Amicus (FOLD)?

POMBILITI has a boxed warning for risk of acute cardiorespiratory failure and infusion reactions; it is contraindicated in pregnancy. According to Amicus, appropriate medical support must be available during infusion and contraception is required.

Will Amicus (FOLD) present pediatric or dose-adjustment data for migalastat at WORLDSymposium 2026?

Yes, poster sessions include an ongoing pediatric open-label study and dose-regimen pharmacokinetic results for patients with severe renal impairment. According to Amicus, the RENEW study and ASPIRE II pediatric trial interim data are being shared.