FABRY

RARE-DISEASE

Structured Monitoring for Early Detection of Fabry Progression

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 <h3 style="text-align:center;">Renal Outcomes After Switching to Agalsidase Beta in Fabry Disease: A Single-Center Analysis</h3></td>
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 <ul>
 <li>Renal involvement is a key feature of FD, characterized by Gb3 deposition in renal cells and progressive nephropathy that can lead to kidney failure by the third to fifth decade in untreated patients with classical mutations (1).</li>
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 <td style="background-color:#DAEBCC;border-color:hsl(0, 0%, 100%);border-width:3px;">
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 <li>Specific therapies have altered FD progression where ERT remains central, with agalsidase alfa, beta, and pegunigalsidase alfa (1).</li>
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 <td style="background-color:#DAEBCC;border-color:hsl(0, 0%, 100%);border-width:3px;">
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 <li>Since recent studies highlighted a dose-dependent effect of agalsidase, showing that higher doses enhance intracellular enzymatic activity, improving Gb3 clearance and slowing nephropathy progression (1).</li>
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 <li>It has been recently suggested that a treatment for classic FD should be considered effective in preserving renal function when it maintains an eGFR decline &lt;1 mL/min/1.73 m2 per year (1).</li>
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 <li>Included subjects were adult patients (≥18 years of age) with genetically determined FD, of both &nbsp;genders, with eGFR between 45 and 90 mL/min/1.73 m2, switched from at least 1 year of stable &nbsp;treatment with the regular dose of agalsidase alfa or migalastat as their primary therapy, to agalsidase beta without any interval (1).</li>
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 <h3 style="text-align:center;">Study objective (1)</h3></td>
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 <td style="border-color:#117B80;border-width:2px;">To evaluate within-patient clinical outcomes, primarily renal outcomes, in adult patients with FD who were switched from agalsidase alfa or migalastat to agalsidase beta due to renal inefficacy of primary therapy, defined by: A linear negative eGFR slope ≥3 mL/min/1.73 m²/year in males ≥2.5 mL/min/1.73 m²/ year in females.</td>
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 <h3 style="text-align:center;">Study design and population (1)</h3></td>
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 <li>This single-centre observational study enrolled 11 adult patients (≥18 years) with genetically confirmed FD and baseline eGFR 45–90 mL/min/1.73 m².</li>
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 <li>Patients meeting the inclusion criteria were adults of both genders who had received stable primary therapy for ≥1 year with agalsidase alfa or migalastat.</li>
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 <ul>
 <li>The cohort was predominantly male (82%), with a mean age of 48.1 ± 11.0 years, and had received primary therapy for a mean duration of 55.3 ± 31.2 months; classical GLA variants were present in 82% and late-onset variants in 18%.</li>
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 <li>Patients meeting inclusion criteria were followed for 24 months after the switch.</li>
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 <h3 style="text-align:center;">Study endpoints (1)</h3></td>
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AEs considered were IARs, dyspnoea, hypertension, gastrointestinal symptoms, rigors, temperature change sensation, fever, headache, rhinitis, flushing, and pruritus. 
 &nbsp;
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 <h3 style="text-align:center;">Study result (1)</h3></td>
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 <td style="background-color:#EAF2D2;" colspan="4">Parameter comparison across therapy initiation, pre-switch, post-switch visits 
 The results compare parameters assessed at primary FD therapy initiation (T0), last pre-switch (T1), and last post-switch visit (T2). No deaths occurred, and no renal, cardiac,or cerebrovascular events were reported during either the pre-switch or post switch periods.</td>
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 <td style="background-color:#78C043;">Parameter</td>
 <td style="background-color:#78C043;">Start of primary Fabry therapy (T0)</td>
 <td style="background-color:#78C043;">Last pre-switch visit (T1)</td>
 <td style="background-color:#78C043;">Last post-switch visit (T2)</td>
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 <td style="background-color:#EAF2D2;">eGFR (mL/min/1.73 m²)</td>
 <td style="background-color:#EAF2D2;">95.5 ± 16.4</td>
 <td style="background-color:#EAF2D2;">67.9 ± 20.0*</td>
 <td style="background-color:#EAF2D2;">67.0 ± 23.6*</td>
 </tr>
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 <td style="background-color:#D0E6BD;">Clinical events, n (%)</td>
 <td style="background-color:#D0E6BD;">0 (0)</td>
 <td style="background-color:#D0E6BD;">0 (0)</td>
 <td style="background-color:#D0E6BD;">0 (0)</td>
 </tr>
 <tr>
 <td style="background-color:#EAF2D2;">Proteinuria (mg/day)</td>
 <td style="background-color:#EAF2D2;">559.1 ± 479.1</td>
 <td style="background-color:#EAF2D2;">645.0 ± 1064.4</td>
 <td style="background-color:#EAF2D2;">513.6 ± 846.6</td>
 </tr>
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 <td style="background-color:#D0E6BD;">LVMI (g/m²)</td>
 <td style="background-color:#D0E6BD;">44.3 ± 12.2</td>
 <td style="background-color:#D0E6BD;">42.5 ± 12.2</td>
 <td style="background-color:#D0E6BD;">513.6 ± 846.6</td>
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 <td style="background-color:#EAF2D2;">Neurologic changes, n (%)</td>
 <td style="background-color:#EAF2D2;">0 (0)</td>
 <td style="background-color:#EAF2D2;">1 (9)</td>
 <td style="background-color:#EAF2D2;">0 (0)</td>
 </tr>
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 <td style="background-color:#D0E6BD;">FD-related symptoms, n (%)</td>
 <td style="background-color:#D0E6BD;">10 (91)</td>
 <td style="background-color:#D0E6BD;">4 (36)*</td>
 <td style="background-color:#D0E6BD;">2 (18)*</td>
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 <td style="background-color:#EAF2D2;">Adverse events, n (%)</td>
 <td style="background-color:#EAF2D2;">Not applicable</td>
 <td style="background-color:#EAF2D2;">2 (18)</td>
 <td style="background-color:#EAF2D2;">2 (18)</td>
 </tr>
 <tr>
 <td style="background-color:#D0E6BD;">Lyso-Gb3 (ng/mL)</td>
 <td style="background-color:#D0E6BD;">20.5 ± 17.9</td>
 <td style="background-color:#D0E6BD;">8.7 ± 8.1</td>
 <td style="background-color:#D0E6BD;">6.9 ± 5.3*</td>
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</figure>
*Significantly different vs T0 (p&lt;0.05).

Pre and post-switch outcome trajectories 
 Switching to agalsidase beta stabilized renal function after prior decline, maintained improvements in FD-related symptoms, sustained reductions in lyso-Gb3, and showed comparable safety, with no increase in adverse events post-switch (1).&nbsp;

*Significantly different vs T0 (p&lt;0.05). Fabry-related symptoms and biomarkers: 
 Most FD-related symptoms improved with primary therapy and remained stable after switching. Plasma lyso-Gb3 levels showed progressive reduction, reaching statistical significance at 24 months versus baseline (p&lt;0.05), although slope differences between treatment periods were not significant (1).

Consistent with these findings, switching to agalsidase beta in patients with progressive renal decline under agalsidase alfa or migalastat resulted in stabilization of eGFR slope, reinforcing the role of dose optimization in ERT to attenuate FD progression and improve renal outcomes (1). 
 &nbsp;
Abbreviation 
 FD – Fabry disease; Gb3 – Globotriaosylceramide; ERT – Enzyme replacement therapy; eGFR – Estimated glomerular filtration rate; CKD-EPI – Chronic Kidney Disease Epidemiology Collaboration; LVMI – Left ventricular mass index; Lyso-Gb3 –&nbsp; Globotriaosylsphingosine; GLA – α-Galactosidase A; T0 – Start of primary Fabry therapy; T1 – Last pre-switch visit; T2 – Last post-switch&nbsp; visit; AE – Adverse event; IARs – Infusion-related reactions; MI – Myocardial infarction 
 &nbsp;

Reference

SPECIALTY-CARE

FABRAZYME

Renal Outcomes After Switching to Agalsidase Beta: A Single-Center Analysis

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 <td style="background-color:#405E7B;">
 Dupilumab for Patients With Allergic Fungal Rhinosinusitis (AFRS)</td>
 </tr>
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</figure>
AFRS is a form of chronic rhinosinusitis characterized by type 2 inflammation and fungal hypersensitivity

aNo participant received a q4w dosage as there were no patients in this weight category. bValues are mean (SD) unless otherwise indicated. cAmong adult patients only, mean (SD) ages is 42.6 (14.2), n=56. dAmericas: Argentina, Canada, and USA. eAsia: China, India, Japan, Saudi Arabia, and Turkey. f8/15 (53.3%) patients in North America were Black or African American. gLund–Mackay (LMK)-CT score of at least 9 (unilateral) or 12 (bilateral) out of 24 (score range score range 0−24; higher score indicates greater sinus opacification). BL, baseline; CI, confidence interval; INCS, intranasal corticosteroids; LMK-CT, Lund-Mackay computed tomography score; LSM, least squares mean; NCS, nasal congestion score; NPS, nasal polyp score; q2w, every 2 weeks; q4w, every 4 weeks; R, randomization; SCS, systemic corticosteroids; SE, standard error. Luong AU, et al. Presented at the American College of Allergy, Asthma, and Immunology (ACAAI) 2025 annual meeting: Orlando, FL, USA; November 6–10, 2025.

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 <td style="background-color:#37783F;">Safety: Treatment-emergent adverse events (TEAEs)</td>
 </tr>
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 </table>
</figure>
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 <td>TEAEs</td>
 <td>Placebo (n=28)b</td>
 <td>Dupilumab (n=33)</td>
 </tr>
 <tr>
 <td style="background-color:#AFC8B2;">Patients with any TEAE</td>
 <td style="background-color:#AFC8B2;">22 (78.6)</td>
 <td style="background-color:#AFC8B2;">23 (69.7)</td>
 </tr>
 <tr>
 <td>Patients with any treatment-emergent SAE</td>
 <td>2 (7.1)</td>
 <td>0</td>
 </tr>
 <tr>
 <td style="background-color:#AFC8B2;">Patients with with any TEAE leading to permanent IMP discontinuationc</td>
 <td style="background-color:#AFC8B2;">1 (3.6)</td>
 <td style="background-color:#AFC8B2;">1 (3.0)</td>
 </tr>
 <tr>
 <td>Patients with any treatment-related TEAE</td>
 <td>3 (10.7)</td>
 <td>9 (27.3)</td>
 </tr>
 <tr>
 <td style="background-color:#AFC8B2;">Patients with any TEAE leading to death</td>
 <td style="background-color:#AFC8B2;">0</td>
 <td style="background-color:#AFC8B2;">0</td>
 </tr>
 <tr>
 <td>&nbsp;</td>
 <td>&nbsp;</td>
 <td>&nbsp;</td>
 </tr>
 <tr>
 <td style="background-color:#AFC8B2;" colspan="3">Overall incidence of TEAEs was similar between placebo and dupilumab</td>
 </tr>
 </tbody>
 </table>
</figure>

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 <td style="background-color:#37783F;">Summary</td>
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 <td>
 In the LIBERTY-AIMS Phase 3 study, dupilumab significantly improved 
 radiographic, endoscopic, and clinical features of AFRS in patients ≥6 years 
 of age, and was generally well tolerated</td>
 </tr>
 </tbody>
 </table>
</figure>

aDupilumab-treated patients had a 29.1% lower risk of receiving SCS and/or undergoing surgery compared to placebo. bOne participant in the placebo group (n = 29) randomized but not treated. cOne participant (3.6%) in the placebo group and 1 participant (3.0%) in the dupilumab group permanently discontinued study intervention due to Sjögren’s syndrome and allergic dermatitis, respectively. CI, confidence interval; IMP, investigational medicinal product; SAE, serious adverse event; SCS, systemic corticosteroids. Luong AU, et al. Presented at the American College of Allergy, Asthma, and Immunology (ACAAI) 2025 annual meeting: Orlando, FL, USA; November 6–10, 2025.

ALLERGIC-FUNGAL-RHINOSINUSITIS

RHINOLOGY

Dupilumab for Patients with AFRS

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Structured Monitoring for Early Detection of Fabry Progression

Renal Outcomes After Switching to Agalsidase Beta: A Single-Center Analysis

Dupilumab for Patients with AFRS

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