Amicus Therapeutics Presents New Long-term Data for both Galafold® (migalastat) and POMBILITI® (cipaglucosidase alfa-atga) + OPFOLDA® (miglustat) at the 22nd Annual WORLDSymposium(TM) 2026
MWN-AI** Summary
Amicus Therapeutics showcased pivotal long-term data for its therapies, Galafold® (migalastat) and POMBILITI® (cipaglucosidase alfa-atga) + OPFOLDA® (miglustat), at the 22nd Annual WORLDSymposium™ 2026. These findings concentrate on treatments for Fabry disease and late-onset Pompe disease, respectively, emphasizing Amicus's commitment to advancing the understanding of these rare diseases.
Dr. Jeff Castelli, Chief Development Officer at Amicus Therapeutics, expressed pride in presenting the new data, which contributes to the accumulating evidence of the efficacy of Galafold and POMBILITI + OPFOLDA. The oral presentations included a comparison of the effectiveness of migalastat versus traditional enzyme replacement therapy in treatment-naïve Fabry patients, along with significant 208-week outcomes for patients with late-onset Pompe disease undergoing treatment combining cipaglucosidase alfa and miglustat.
Among the notable presentations, Peter Nordbeck from the University of Wuerzburg shared data on the real-world efficacy of migalastat, while Tahseen Mozaffar from UC Irvine discussed the promising muscle function and biomarker outcomes for Pompe disease. Various poster sessions highlighted the lived experiences of Fabry patients, ongoing clinical evaluations, and the unique methodologies used to assess patient-reported outcomes across treatment programs.
Galafold is an oral treatment for Fabry disease that stabilizes the body's enzyme, helping to clear disease-related substrate. Meanwhile, POMBILITI and OPFOLDA, a two-component therapy for late-onset Pompe disease, demonstrate promising results for patients who have not responded adequately to previous enzyme replacement therapies.
These findings collectively reinforce the importance of continuous research and collaboration in uncovering new insights for individuals afflicted with rare diseases. Amicus Therapeutics remains dedicated to delivering transformative therapies that can improve patients' quality of life.
MWN-AI** Analysis
Amicus Therapeutics (Nasdaq: FOLD) recently presented compelling long-term data for Galafold® (migalastat) and POMBILITI® (cipaglucosidase alfa-atga) + OPFOLDA® (miglustat) during the 22nd Annual WORLDSymposium™. This information is crucial for potential investors as it underlines the ongoing efficacy and safety of these treatments for Fabry and Pompe diseases, respectively.
Galafold® has demonstrated promising results, particularly in patients who are treatment-naïve, providing a comparative advantage over conventional enzyme replacement therapies (ERT). The ability of Galafold to stabilize the alpha-Gal A enzyme shows a valuable mechanism that could position Amicus favorably against competitors in the realm of rare disease therapies. With an estimated 35-50% of Fabry patients eligible for treatment, the market opportunity remains substantial.
For patients with late-onset Pompe disease, the combination of POMBILITI and OPFOLDA has been highlighted as offering superior muscle function and biomarker outcomes over longer durations. This could drastically improve patient quality of life and potentially underscore the drug’s therapeutic value in the market, which could lead to increased adoption by healthcare providers once further validation from ongoing studies is confirmed.
Investors should closely watch the regulatory landscape and market response as Amicus navigates the complexities associated with patient safety reports and the need for continued studies to confirm long-term benefits. The current data supports a bullish outlook, yet concerns over safety issues, particularly with thromboembolic events and infusion-related reactions, cannot be overlooked.
With a pipeline focused on transformative therapies for rare diseases, Amicus Therapeutics presents an intriguing opportunity for investors, especially as more long-term outcomes emerge. A proactive approach to monitoring ongoing clinical trials and market trends will allow investors to make informed decisions moving forward.
**MWN-AI Summary and Analysis is based on asking OpenAI to summarize and analyze this news release.
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
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FAQ**
How do the new data presented by Amicus Therapeutics Inc. FOLD at the WORLDSymposium enhance the understanding of Galafold's effectiveness for patients with Fabry disease compared to traditional enzyme replacement therapies?
What insights do the 208-week outcomes of POMBILITI + OPFOLDA treatment in late-onset Pompe disease provide regarding long-term muscle function and biomarker improvements, as discussed by Amicus Therapeutics Inc. FOLD?
How does Amicus Therapeutics Inc. FOLD plan to address the safety concerns raised in the clinical presentations regarding their therapies, particularly related to adverse reactions in patients with Fabry and Pompe diseases?
In light of the results shared by Amicus Therapeutics Inc. FOLD, what are the implications for future clinical trials and regulatory approval processes for Galafold and POMBILITI + OPFOLDA in treating rare diseases?
**MWN-AI FAQ is based on asking OpenAI questions about Amicus Therapeutics Inc. (NASDAQ: FOLD).
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