Elfabrio (pegunigalsidase)
Research Synopsis
Elfabrio (pegunigalsidase)
Development and Analytical Characterization of Pegunigalsidase Alfa, a Chemically Cross-Linked Plant Recombinant Human α-Galactosidase-A for Treatment of Fabry Disease.
London, UK
2 hours ago
1 Received
- Current enzyme replacement therapy for Fabry disease using α-Galactosidase A is showing continuous patient deterioration.
- A study evaluated how covalently bonding α-Galactosidase A subunits with PEG cross-linkers enhances protein stability and distribution.
- The modified enzyme, pegunigalsidase alfa, is being tested in phase III clinical trials and may prove more effective than existing treatments.
$100 - $150
Hourly Rate
Pegunigalsidase alfa, a novel PEGylated enzyme replacement therapy for Fabry disease, provides sustained plasma concentrations and favorable pharmacodynamics: A 1-year Phase 1/2 clinical trial.
London, UK
2 hours ago
1 Received
- Pegunigalsidase alfa is a new enzyme replacement therapy for Fabry disease designed to improve the duration of its effects and lower the risk of immune reactions compared to existing treatments.
- In a study with symptomatic adults, dosage levels of 0.2, 1.0, and 2.0 mg/kg were tested over 12 months, showing significant reductions in harmful substances in kidneys and stable renal function.
- The therapy demonstrated a range of plasma half-lives and low rates of severe side effects, with most adverse events being mild or moderate, suggesting it could be a significant improvement in treating Fabry disease.
$100 - $150
Hourly Rate
Developments in the treatment of Fabry disease.
London, UK
2 hours ago
1 Received
- Enzyme replacement therapy (ERT) with recombinant α-galactosidase A has been used for over 15 years to treat Fabry disease, but many patients still experience serious complications despite the treatment.
- A new oral treatment, chaperone therapy (Migalastat), is only approved for patients with specific mutations and works by enhancing the activity of the mutated enzyme.
- Research is ongoing for additional therapies, including second-generation ERTs and substrate reduction therapies, as well as promising mRNA and gene-based treatments, which may provide new options for patients in the future.
$100 - $150
Hourly Rate
Therapeutic advances in Fabry disease: The future awaits.
London, UK
2 hours ago
1 Received
- * Current treatments include enzyme replacement therapy (ERT), which primarily benefits kidney function but has limitations like the formation of anti-drug antibodies, and chaperone therapy, which is only applicable for certain mutations.
- * A range of new therapies are being explored, such as pegylated α-GAL, gene therapy, mRNA therapy, and substrate reduction therapy, all aiming to improve outcomes for individuals with Fabry disease.
$100 - $150
Hourly Rate
Fabry Disease Therapy: State-of-the-Art and Current Challenges.
London, UK
2 hours ago
1 Received
- Fabry disease (FD) is a genetic disorder caused by a lack of the enzyme α-galactosidase A due to mutations in the gene, leading to the buildup of harmful substances in the body.
- Current treatments include enzyme replacement therapy (ERT) with agalsidase alfa and beta, and the chaperone migalastat, but unresolved issues remain regarding their effectiveness, dosing, and timing.
- New therapies under research, such as innovative ERT forms, substrate reduction therapy, mRNA therapy, and gene therapy, aim to improve treatment outcomes since existing therapies do not completely resolve the disease's symptoms.
$100 - $150
Hourly Rate
[The treatment for Fabry disease: focus on agalsidase alpha and beta].
London, UK
2 hours ago
1 Received
- Fabry disease (FD) is an inherited condition linked to the X chromosome, caused by a lack of the enzyme α-galactosidase A, leading to harmful substance buildup and various health issues affecting multiple body systems.
- While hemizygous males typically experience severe symptoms (neurological pain, skin rashes, kidney problems, and heart issues), heterozygous females may have a range of symptoms from mild to severe.
- Treatment options include enzyme replacement therapy (ERT) with agalsidase alfa or beta, proven effective in improving symptoms, and migalastat, an oral therapy that helps certain enzyme mutations; both approaches highlight a need for tailored therapies based on individual patient profiles.
$100 - $150
Hourly Rate
Anderson-Fabry disease: No histological signs of pathological accumulation in arterial and venous endothelium during pegunigalsidase alfa therapy.
London, UK
2 hours ago
1 Received
$100 - $150
Hourly Rate
Pre-existing anti-drug antibodies in Fabry disease show less affinity for pegunigalsidase alfa.
London, UK
2 hours ago
1 Received
- * Pull-down experiments indicated that PEGylation of PRX-102 masks certain epitopes, which likely contributes to the reduced ADA affinity and a notable decrease in inhibitory effects on PRX-102.
- * Our findings suggest that switching from traditional treatments to PRX-102 could be beneficial for patients with pre-existing ADAs, and this transition is currently being tested in clinical trials
$100 - $150
Hourly Rate
Pegunigalsidase Alfa-iwxj.
London, UK
2 hours ago
1 Received
$100 - $150
Hourly Rate
Safety and efficacy of pegunigalsidase alfa in patients with Fabry disease who were previously treated with agalsidase alfa: results from BRIDGE, a phase 3 open-label study.
London, UK
2 hours ago
1 Received
- Pegunigalsidase alfa is a new enzyme-replacement therapy approved for treating Fabry disease, evaluated for 12 months in a phase 3 study involving adults previously treated with agalsidase alfa.
- The study found that pegunigalsidase alfa was well-tolerated, with most adverse events being mild or moderate, and showed a significant reduction in the annual decline of kidney function compared to prior treatment.
- Overall, a portion of patients developed antibodies against the treatment, but this did not negatively affect their kidney function, suggesting pegunigalsidase alfa could be a promising option for Fabry disease management.
$100 - $150
Hourly Rate
Characterization of pre-existing anti-PEG and anti-AGAL antibodies towards PRX-102 in patients with Fabry disease.
London, UK
2 hours ago
1 Received
- Polyethylene glycol (PEG)ylated drugs, like pegunigalsidase-alfa (PRX-102), are used in enzyme replacement therapy for Fabry disease, benefiting from reduced drug clearance and immunogenicity.
- Exposure to PEG from lifestyle products and vaccines can lead to anti-PEG antibody production, as seen in a healthy male post-mRNA vaccination for COVID-19.
- In a study of 102 Fabry disease patients, those without anti-AGAL antibodies had higher enzyme activity and less inhibition from both anti-AGAL and anti-PEG antibodies, indicating that PRX-102 may offer better treatment outcomes compared to current enzyme therapies.
$100 - $150
Hourly Rate
Long-term safety and efficacy of pegunigalsidase alfa: A multicenter 6-year study in adult patients with Fabry disease.
London, UK
2 hours ago
1 Received
- Fabry disease is a rare condition caused by mutations in the GLA gene, leading to a deficiency of the enzyme α-galactosidase, and this study evaluates the long-term safety and effectiveness of a new treatment called pegunigalsidase alfa.
- *The study involved 15 adults with Fabry disease who received bi-weekly infusions of this enzyme replacement therapy for up to 60 months, with most adverse events being mild to moderate.
- *Results showed a significant, continuous decrease in a specific plasma biomarker and stability in kidney and heart function, indicating the treatment has favorable long-term effects.*
$100 - $150
Hourly Rate
Pegunigalsidase alfa: a novel, pegylated recombinant alpha-galactosidase enzyme for the treatment of Fabry disease.
London, UK
2 hours ago
1 Received
- - Fabry disease, an X-linked genetic disorder caused by a deficiency in the enzyme α-galactosidase A, leads to a range of health issues, and since 2001, enzyme replacement therapy (ERT) has been the primary treatment, although it has drawbacks like quick clearance and immune reactions.
- - Pegunigalsidase alfa, a new PEGylated version of the enzyme, improves stability, half-life, and reduces immune responses, showing promising results in clinical trials for renal function and GB3 clearance.
- - Although recent phase 3 trials (BRIDGE, BRIGHT, and BALANCE) indicate that pegunigalsidase alfa is effective and safe, caution is needed in interpreting
$100 - $150
Hourly Rate
Head-to-head trial of pegunigalsidase alfa versus agalsidase beta in patients with Fabry disease and deteriorating renal function: results from the 2-year randomised phase III BALANCE study.
London, UK
2 hours ago
1 Received
- Pegunigalsidase alfa is a new enzyme replacement therapy compared to agalsidase beta for treating Fabry disease, evaluated through the BALANCE trial.
- In the study, 77 adult patients were randomly assigned to receive either treatment every two weeks for two years, focusing on eGFR slope differences to measure effectiveness.
- Results showed that pegunigalsidase alfa was not inferior to agalsidase beta in terms of eGFR decline, with fewer adverse events and infusion reactions for the new treatment.
$100 - $150
Hourly Rate
Comment to: Head-to-head trial of pegunigalsidase alfa versus agalsidase beta in patients with Fabry disease and deteriorating renal function: results from the 2-year randomised phase III BALANCE study-determination of immunogenicity.
London, UK
2 hours ago
1 Received
$100 - $150
Hourly Rate
Response to commentary: Head-to-head trial of pegunigalsidase alfa versus agalsidase beta in patients with Fabry disease and deteriorating renal function: results from the 2-year randomised phase III BALANCE study - determination of immunogenicity.
London, UK
2 hours ago
1 Received
$100 - $150
Hourly Rate
[What is confirmed in the treatment of Fabry's disease?].
London, UK
2 hours ago
1 Received
- Fabry's disease is a rare genetic disorder linked to the X chromosome, caused by low levels of the enzyme alpha-galactosidase A (AGAL), leading to harmful build-up of globotriaosylceramide in the body's tissues.
- The condition significantly affects life expectancy, reducing it by approximately 10 years for females and 20 years for males due to serious complications like kidney failure, heart problems, and strokes.
- Current treatments include enzyme replacement therapy with specific medications administered every two weeks or an oral therapy for certain gene mutations, with ongoing discussions about new guidelines and research to enhance future treatment options.
$100 - $150
Hourly Rate
Establishing Treatment Effectiveness in Fabry Disease: Observation-Based Recommendations for Improvement.
London, UK
2 hours ago
1 Received
- Fabry disease (FD) results from mutations in a specific gene that lead to a deficiency in the enzyme α-galactosidase A, causing harmful substrate accumulation, with symptoms varying widely between male and female patients.
- Although FD therapies like enzyme replacement therapy (ERT) and chaperone therapy have been available for around 20 years, there is still limited evidence supporting their long-term effectiveness due to diverse patient populations and inconsistencies in study designs.
- To improve future research on FD treatments, the review suggests better patient matching, international collaboration, and standardized approaches for evaluating treatment effectiveness, including clear recommendations for study outcomes and duration.
$100 - $150
Hourly Rate
A phase III, open-label clinical trial evaluating pegunigalsidase alfa administered every 4 weeks in adults with Fabry disease previously treated with other enzyme replacement therapies.
London, UK
2 hours ago
1 Received
- Pegunigalsidase alfa is a new enzyme replacement therapy for Fabry disease, showing a longer half-life and administered every 4 weeks instead of the typical 2-week schedule.
- The BRIGHT study involved 30 adult patients who switched from another ERT to pegunigalsidase alfa, revealing good safety results with mostly mild side effects and no new anti-drug antibodies.
- Although the treatment showed acceptable tolerance, more research is needed due to the small sample size, but it suggests that this 4-week regimen could be a viable new option for managing Fabry disease.
$100 - $150
Hourly Rate