- |||||||||| Review, Journal: What is confirmed in the treatment of Fabry's disease? (Pubmed Central) - Dec 10, 2024
TAKE-HOME MESSAGE: Treatment with 2 mg/kg pegunigalsidase alfa every 4 weeks could offer a new treatment option for patients with Fabry disease. The treatment comprises enzyme replacement therapy (ERT), agalsidase alfa, 0.2?mg/kg body weight (BW), agalsidase beta 1.0?mg/kg BW or pegunigalsidase alfa 1.0?mg/kg BW every 2
- |||||||||| Galafold (migalastat) / Amicus, Elfabrio (pegunigalsidase alfa-iwxj) / Protalix, Chiesi, Replagal (agalsidase alfa) / Samaritan Pharma, Takeda
Review, Journal: Establishing Treatment Effectiveness in Fabry Disease: Observation-Based Recommendations for Improvement. (Pubmed Central) - Sep 14, 2024
We recommend international collaboration and harmonization, facilitated by an independent FD registry. We propose a stepwise approach for evaluating the effectiveness of novel treatments, including recommendations for surrogate outcomes and required study duration.
- |||||||||| Replagal (agalsidase alfa) / Samaritan Pharma, Takeda
Observational data, Retrospective data, Journal: Agalsidase alfa long-term effect on left ventricular hypertrophy in Fabry disease. (Pubmed Central) - Jun 22, 2024
Our results are in line with previous literature of comparable FD populations and probably represent the first study of its kind in Argentina. We here highlight the importance of cardiac morphometric stability as a positive outcome of ERT.
- |||||||||| Ibrance (palbociclib) / Pfizer, Spinraza (nusinersen) / Biogen, Ionis, Replagal (agalsidase alfa) / Samaritan Pharma, Takeda
Retrospective data, Journal: Judicialization of high priced medications in Argentina: quali-quantitative study (Pubmed Central) - Jun 22, 2024
The rulings were mostly in favor of the plaintiff, and access times to the medication took a long time. The mass media anticipated the judicial processes.
- |||||||||| Replagal (agalsidase alfa) / Samaritan Pharma, Takeda
New P3 trial: A Study of REPLAGAL (EUDRACT) - Jun 11, 2024
P3, N=20, ,
- |||||||||| Replagal (agalsidase alfa) / Samaritan Pharma, Takeda
Trial completion date, Trial primary completion date: A Study of Replagal in Children and Adults With Fabry Disease in India (clinicaltrials.gov) - May 7, 2024
P4, N=5, Active, not recruiting,
The mass media anticipated the judicial processes. Trial completion date: Nov 2024 --> Nov 2026 | Trial primary completion date: Oct 2024 --> Oct 2026
- |||||||||| Galafold (migalastat) / Amicus, Fabrazyme (agalsidase beta) / Sanofi, Replagal (agalsidase alfa) / Samaritan Pharma, Takeda
NEWLY DETECTED MISSENSE VARIANT IN FABRY DISEASE (SAT-164; Exhibition Hall and Main Foyer) - Mar 8, 2024 - Abstract #ISNWCN2024ISN_WCN_1257;
It is important to describe new mutations to facilitate genetic diagnosis. In this case, FD was confirmed with a renal biopsy although the patient had mild proteinuria, identifying the VUS as a pathogenic variable and avoiding an endomyocardial biopsy.
- |||||||||| Replagal (agalsidase alfa) / Samaritan Pharma, Takeda
JEJUNAL DIVERTICULOSIS AND ZEBRA BODIES IN FABRY DISEASE: A CASE REPORT (SAT-159; Exhibition Hall and Main Foyer) - Mar 8, 2024 - Abstract #ISNWCN2024ISN_WCN_1252;
Abdominal pain, nausea, vomiting, diarrhea or constipation occur in approximately 20-70% of patients. These symptoms are caused by the deposition of glycosphingolipids in the autonomic intestinal ganglia and mesenteric blood vessels, leading to intestinal dysmotility, autonomic dysfunction, vasculopathy and myopathy.Small bowel diverticula are typically asymptomatic but can rarely lead to severe and life threatening complications.
- |||||||||| Humanistic Burden of Fabry Disease and Associated Utility Values () - Mar 8, 2024 - Abstract #ISPOR2024ISPOR_2023;
The results suggest utility values increase with ERT treatment, but patient burden has remained stable over time. Long-term data with existing therapies may provide additional insights on outcomes including pain, disease severity, and quality of life.
- |||||||||| Replagal (agalsidase alfa) / Samaritan Pharma, Takeda
Journal: Complement activation and cellular inflammation in Fabry disease patients despite enzyme replacement therapy. (Pubmed Central) - Feb 6, 2024
Enzyme replacement therapy (ERT) with agalsidase-alfa or -beta is one of the main treatment options facilitating cellular Gb3 clearance...In addition, our data suggest kidney cell-associated production of cytokines, which have a strong potential to drive renal damage. Thus, chronic inflammation as a driver of organ damage in FD seems to proceed despite ERT and may prove useful as a target to cope with progressive organ damage.
- |||||||||| Replagal (agalsidase alfa) / Samaritan Pharma, Takeda
Trial completion: A Study of REPLAGAL (clinicaltrials.gov) - Jan 21, 2024
P3, N=20, Completed,
Thus, chronic inflammation as a driver of organ damage in FD seems to proceed despite ERT and may prove useful as a target to cope with progressive organ damage. Recruiting --> Completed
- |||||||||| Voxzogo (vosoritide) / BioMarin, Replagal (agalsidase alfa) / Samaritan Pharma, Takeda
To Treat or Not to Treat: Dilemmas for Rare Diseases Care in A New Therapeutic Era (MTCC - 701) - Dec 23, 2023 - Abstract #ACMG2024ACMG_116;
Dr. Robert J. Hopkin, clinical geneticist with vast experience in both research and clinical care for FD will present a perspective in favor of early administration for ERT in asymptomatic non-classical Het-Ind.Learning Objectives Evaluate the reported direct and indirect benefits of the administration of biologic agents such as vosoritide Review the potential long-term side effects of recently approved therapies Explain the current recommendations for ERT in classical and non-classical hemizygous FD patients Identify the possible risks of ERT administration in asymptomatic non-classical heterozygous FD patients
- |||||||||| venglustat (GZ402671) / Sanofi
Trial primary completion date: CARAT: A Study to Evaluate the Effect of Venglustat Tablets on Left Ventricular Mass Index in Male and Female Adult Participants With Fabry Disease (clinicaltrials.gov) - Dec 22, 2023
P3, N=90, Recruiting,
Robert J. Hopkin, clinical geneticist with vast experience in both research and clinical care for FD will present a perspective in favor of early administration for ERT in asymptomatic non-classical Het-Ind.Learning Objectives Evaluate the reported direct and indirect benefits of the administration of biologic agents such as vosoritide Review the potential long-term side effects of recently approved therapies Explain the current recommendations for ERT in classical and non-classical hemizygous FD patients Identify the possible risks of ERT administration in asymptomatic non-classical heterozygous FD patients Trial primary completion date: Jun 2025 --> Dec 2025
- |||||||||| Replagal (agalsidase alfa) / Samaritan Pharma, Takeda
Trial completion date, Trial primary completion date: A Study of REPLAGAL (clinicaltrials.gov) - Nov 30, 2023
P3, N=20, Recruiting,
Trial primary completion date: Jun 2025 --> Dec 2025 Trial completion date: May 2024 --> Jan 2024 | Trial primary completion date: May 2024 --> Jan 2024
- |||||||||| Firazyr (icatibant) / Takeda, Replagal (agalsidase alfa) / Samaritan Pharma, Takeda
RWD From Patient Registry for Rare Diseases and Its Uses in HTA Process in Brazil () - Aug 25, 2023 - Abstract #ISPOREU2023ISPOR_EU_170;
The new submissions (2022) did not include novel RCT data, thus including FOS and IOS data, the clinical dossier added long-term effectiveness and safety data. Agalsidase-alfa was recently incorporated by Conitec-SUS and Icatibant HTA-process is ongoing.Lessons Learned: RWE published using patient registry with local data added value on the long-term effectiveness and safety that is important for HTA decision making.Stakeholder perspective: Industry
- |||||||||| Replagal (agalsidase alfa) / Samaritan Pharma, Takeda
A Novel Multiplex approach for determining anti-drug antibodies in Fabry Disease (Exhibitions Hall (ICC)) - Jul 11, 2023 - Abstract #SSIEM2023SSIEM_517;
Results Pilot analysis (n=60 FD patients) using commercial ERT Agalsidase alfa as bait detected a positive response for IgG1-3 in 34-38% and IgG4 in 27% of patients...Conclusions We have developed a novel methodology for determining ADA response to ERT in FD patients. This assay can also identify differential ADA response depending on the glycosylation of the
- |||||||||| Replagal (agalsidase alfa) / Samaritan Pharma, Takeda
Enrollment change, Trial completion date, Trial termination, Trial primary completion date: A Study of Replagal in Treatment-na (clinicaltrials.gov) - May 6, 2023
P3, N=17, Terminated,
Long-term observation of electrocardiogram changes might be useful for determining the efficacy of ERT in this case. N=45 --> 17 | Trial completion date: Dec 2025 --> Dec 2022 | Recruiting --> Terminated | Trial primary completion date: Dec 2025 --> Dec 2022; Study closed due to enrolment challenges, not for any safety issues
- |||||||||| Replagal (agalsidase alfa) / Samaritan Pharma, Takeda
Enrollment closed: A Study of Replagal in Children and Adults With Fabry Disease in India (clinicaltrials.gov) - Mar 29, 2023
P4, N=5, Active, not recruiting,
N=45 --> 17 | Trial completion date: Dec 2025 --> Dec 2022 | Recruiting --> Terminated | Trial primary completion date: Dec 2025 --> Dec 2022; Study closed due to enrolment challenges, not for any safety issues Recruiting --> Active, not recruiting
- |||||||||| Galafold (migalastat) / Amicus
FABRY NEPHROPATHY: SWITCH FROM ENZYME REPLACEMENT THERAPY TO ORAL CHAPERONE MIGALASTAT () - Mar 28, 2023 - Abstract #ISNWCN2023ISN_WCN_1159;
Moreover, being an oral therapy allows more sustained and stable ?-gal-A levels than ERT. In conclusion, due to its proven efficacy (reduction of left ventricular mass index, and stabilization of renal function and disease biomarkers), the oral administration regimen and the limited therapeutic options to date, migalastat is a valid alternative for switching from ERT or initiate therapy in patients with FD and amenable mutations.
- |||||||||| Clinical, Review, Journal: Fabry Disease: current & novel therapeutic strategies. A narrative review. (Pubmed Central) - Mar 27, 2023
In conclusion, due to its proven efficacy (reduction of left ventricular mass index, and stabilization of renal function and disease biomarkers), the oral administration regimen and the limited therapeutic options to date, migalastat is a valid alternative for switching from ERT or initiate therapy in patients with FD and amenable mutations. The therapeutic landscape in FD appears to be actively expanding with more treatment options expected to become available in the near future, allowing for a more personalized approach in FD patients.
- |||||||||| Fabrazyme (agalsidase beta) / Sanofi, Replagal (agalsidase alfa) / Samaritan Pharma, Takeda
A Cost Analysis on Complications of Fabry Disease Patients Treated with Agalsidase Alfa and Agalsidase Beta in Colombia () - Mar 9, 2023 - Abstract #ISPOR2023ISPOR_992;
The therapeutic landscape in FD appears to be actively expanding with more treatment options expected to become available in the near future, allowing for a more personalized approach in FD patients. The results suggest that the treatment of Fabry Disease with agalsidase beta is associated with better health outcomes, given a lower occurrence of clinical complications, as well as the potential avoided costs and reduced use of healthcare resources for the management of these events.
- |||||||||| Fabrazyme (agalsidase beta) / Sanofi, Replagal (agalsidase alfa) / Samaritan Pharma, Takeda
Journal: Assessment and impact of dose escalation on anti-drug antibodies in Fabry disease. (Pubmed Central) - Dec 27, 2022
Dose escalation results in a heterogeneous, unpredictable ADA response, with more than a quarter of all treatment switchers succeeding in ADA saturation. Longitudinal ADA measurements are required to assess the individual risk of affected patients.
- |||||||||| Replagal (agalsidase alfa) / Samaritan Pharma, Takeda
Enrollment open: A Study of Replagal in Children and Adults With Fabry Disease in India (clinicaltrials.gov) - Nov 3, 2022
P4, N=5, Recruiting,
The update of the review on the effects of switching from agalsidase beta to alfa showed, in comparison to the previous review, an increased number of clinical events, a significant loss of renal function, and an increase in lyso Gb-3 levels, underscoring the importance of dose in the treatment of FD. Not yet recruiting --> Recruiting
- |||||||||| Replagal (agalsidase alfa) / Samaritan Pharma, Takeda
Journal: Renoprotective Effect of Agalsidase Alfa: A Long-Term Follow-Up of Patients with Fabry Disease. (Pubmed Central) - Aug 27, 2022
Patients with classic Fabry disease had similar rates of eGFR decline irrespective of baseline proteinuria; only one patient with non-classic Fabry disease had high baseline proteinuria, preventing meaningful comparisons between groups. In this analysis, baseline proteinuria significantly impacted the rate of eGFR decline in the overall population, suggesting that early treatment with good proteinuria control may be associated with renoprotective effects.
- |||||||||| pegunigalsidase alfa (PRX-102) / Protalix, Chiesi
Journal: Pre-existing anti-drug antibodies in Fabry disease show less affinity for pegunigalsidase alfa. (Pubmed Central) - Aug 24, 2022
Enzyme-uptake experiments in AGAL-deficient EA.hy926 cells demonstrated less effects of ADAs on cellular PRX-102 uptake compared with agalsidase beta. We conclude that due to the reduced affinity of pre-existing ADAs against agalsidase-alfa or -beta, ADA-affected patients might benefit from a therapy switch to PRX-102, which is currently evaluated in clinical trials.
- |||||||||| Replagal (agalsidase alfa) / Samaritan Pharma, Takeda
Enrollment open: A Study of REPLAGAL (clinicaltrials.gov) - Jul 18, 2022
P3, N=20, Recruiting,
We conclude that due to the reduced affinity of pre-existing ADAs against agalsidase-alfa or -beta, ADA-affected patients might benefit from a therapy switch to PRX-102, which is currently evaluated in clinical trials. Not yet recruiting --> Recruiting
- |||||||||| Galafold (migalastat) / Amicus, Fabrazyme (agalsidase beta) / Sanofi, Replagal (agalsidase alfa) / Samaritan Pharma, Takeda
Journal: The treatment for Fabry disease: focus on agalsidase alpha and beta (Pubmed Central) - Apr 14, 2022
Two different products, agalsidase alfa and agalsidase beta, have been commercially available in Europe for 20 years and they are both indicated for long-term ERT...A multitude of therapies are now under investigation in various phases of clinical trials. These include pegylated form of α-Gal (pegunigalsidase alpha), gene therapy (both in-vivo and ex-vivo methods), mRNA therapy (inducing production of α-Gal) and substrate reduction therapy (inhibitors of glucosylceramide synthase leading to reduction of Gb-3).
- |||||||||| Replagal (agalsidase alfa) / Samaritan Pharma, Takeda
Trial initiation date: A Study of REPLAGAL (clinicaltrials.gov) - Apr 1, 2022
P3, N=20, Not yet recruiting,
Not yet recruiting --> Recruiting Initiation date: Dec 2021 --> May 2022
- |||||||||| Replagal (agalsidase alfa) / Samaritan Pharma, Takeda
Trial completion date, Trial primary completion date: A Study of Replagal in Children and Adults With Fabry Disease in India (clinicaltrials.gov) - Feb 23, 2022
P4, N=5, Not yet recruiting,
This indicates that a biomarker cut-off could potentially be a decision tool for treatment initiation in women with Fabry disease. Trial completion date: Jan 2024 --> Apr 2024 | Trial primary completion date: Dec 2023 --> Mar 2024
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