Abstract Details

Title Real-world Experience of Switching Treatments from Alglucosidase Alfa to Avalglucosidase Alfa for Patients Enrolled in the Pompe Registry

Topic Neuromuscular and Clinical Neurophysiology (EMG)

Presentation(s) P3 - Poster Session 3 (5:30 PM-6:30 PM)

Poster/Presentation
Number 11-007

Objective

The aim of this analysis is to characterize the demographic and clinical characteristics of infantile-onset (IOPD) and late-onset Pompe disease (LOPD) patients who switched from alglucosidase alfa (ALG) to avalglucosidase alfa (AVA) enrolled in the ongoing international, observational, voluntary Pompe Registry (NCT00231400).

Background

AVA, a recombinant human acid α-glucosidase enzyme replacement therapy, has received marketing authorization in several countries for IOPD and/or LOPD; US approval: August 2021 for LOPD patients aged ≥1 year.

Design/Methods

For this analysis, switch patients were defined as having ≥1 ALG record immediately pre-switch to AVA. Demographic and treatment histories were summarized. Respiratory, ambulatory, and biomarker data were assessed pre- and post-switch for LOPD (IOPD was excluded due to small cohort at data cut).

Results

As of August 4, 2023, 145 LOPD+IOPD switch patients were identified (LOPD, 125 [86.2%]; IOPD, 20 [13.8%]). LOPD: United States, 105 (84.0%); EMEA, 18 (14.4%); JAPAC, 2 (1.6%); female, 60 (48.0%). IOPD: United States, 7 (35.0%); EMEA, 12 (60.0%); JAPAC, 1 (5.0%); female, 12 (60.0%). Patients switched at mean±SD age of 47.4±21.48 (range: 1.0–87.1) years for LOPD and 10.3±5.21 (range: 3.0–20.6) years for IOPD. Most had ≥5 years’ experience on ALG pre-switch (LOPD, 72 [57.6%]; IOPD, 13 [65.0%]). For LOPD, last assessments pre-switch (mean±SD) were upright forced vital capacity % predicted: 60.2±23.98 (n=100), 6-minute walk test: 339.3±157.01 m (n=58), urine glucose tetrasaccharide/hexose tetrasaccharide: 8.2±14.55 mmol/mol creatinine (n=74), and serum creatine kinase: 499.3±399.70 U/L (n=102). Mean changes among LOPD patients with both pre- and up to 1-year post-switch assessments showed stabilization in respiratory and ambulatory function and biomarker improvement.

Conclusions

The Pompe Registry continues to accrue data for patients switching from ALG to AVA, which will support our understanding of AVA’s effectiveness on respiratory and ambulatory outcomes and biomarker levels in the real-world. [On behalf of the Pompe Registry Sites] Funding: Sanofi.

Authors/Disclosures
Mazen M. Dimachkie, MD, FAAN (University of Kansas Medical Center) PRESENTER	Dr. Dimachkie has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Sanofi. Dr. Dimachkie has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Octapharma. Dr. Dimachkie has received personal compensation in the range of $500-$4,999 for serving as a Consultant for ArgenX. Dr. Dimachkie has received personal compensation in the range of $0-$499 for serving as a Consultant for Catalyst. Dr. Dimachkie has received personal compensation in the range of $0-$499 for serving as a Consultant for Takeda. Dr. Dimachkie has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Medlink. Dr. Dimachkie has received personal compensation in the range of $500-$4,999 for serving as a Consultant for CSL Behring. Dr. Dimachkie has received personal compensation in the range of $0-$499 for serving as a Consultant for Astellas. Dr. Dimachkie has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Janssen. Dr. Dimachkie has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Dianthus. Dr. Dimachkie has received personal compensation in the range of $0-$499 for serving as a Consultant for TACT / Treat NMD. Dr. Dimachkie has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Cabaletta Bio. Dr. Dimachkie has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Clinical Neurological Society of America, Inc . Dr. Dimachkie has received personal compensation in the range of $0-$499 for serving as a Consultant for Ig Society, Inc. Dr. Dimachkie has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Abcuro. Dr. Dimachkie has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Amicus. Dr. Dimachkie has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Horizon/Immunovant. Dr. Dimachkie has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for EMD Serono. Dr. Dimachkie has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Ipsen. Dr. Dimachkie has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Nuvig. Dr. Dimachkie has received personal compensation in the range of $500-$4,999 for serving as a Consultant for ��ɫ�� (AAN). Dr. Dimachkie has received personal compensation in the range of $500-$4,999 for serving as a Consultant for American Association of Neuromuscular and Electrodiagnostic Medicine (AANEM). Dr. Dimachkie has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Annexon Biosciences. Dr. Dimachkie has received personal compensation in the range of $500-$4,999 for serving as a Consultant for AstraZeneca. Dr. Dimachkie has received personal compensation in the range of $0-$499 for serving as a Consultant for Candid Therapeutics. Dr. Dimachkie has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Creyon Bio, Inc.. Dr. Dimachkie has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Nuvig Therapeutics. Dr. Dimachkie has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for UCB Biopharma. Dr. Dimachkie has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Vertex Pharmaceuticals. Dr. Dimachkie has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Fortrea. Dr. Dimachkie has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Kriya Therapeutics. The institution of Dr. Dimachkie has received research support from NIH. The institution of Dr. Dimachkie has received research support from The Myositis Association. The institution of Dr. Dimachkie has received research support from Biosensics. Dr. Dimachkie has received intellectual property interests from a discovery or technology relating to health care. Dr. Dimachkie has received intellectual property interests from a discovery or technology relating to health care. Dr. Dimachkie has received intellectual property interests from a discovery or technology relating to health care. Dr. Dimachkie has received publishing royalties from a publication relating to health care. Dr. Dimachkie has received publishing royalties from a publication relating to health care.
Priya Kishnani, MD (Duke University)	Priya Kishnani, MD has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Sanofi/Genzyme. Priya Kishnani, MD has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Amicus Therapeutics. Priya Kishnani, MD has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Maze Therapeutics. Priya Kishnani, MD has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for JCR Pharmaceutical. Priya Kishnani, MD has received personal compensation in the range of $500,000-$999,999 for serving as a Consultant for Asklepios Biopharmaceutical Inc. (AskBio). Priya Kishnani, MD has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for Sanofi/Genzyme. Priya Kishnani, MD has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for Amicus Therapeutics. Priya Kishnani, MD has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for Baebies, Inc.. The institution of Priya Kishnani, MD has received research support from Sanofi/Genzyme. Priya Kishnani, MD has received research support from Amicus Therapeutics. Priya Kishnani, MD has received intellectual property interests from a discovery or technology relating to health care. Priya Kishnani, MD has received intellectual property interests from a discovery or technology relating to health care.
Mary-Alice Abbott (Baystate Medical Center)	No disclosure on file
Antonio Toscano, MD	Dr. Toscano has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Sanofi Genzyme.
Meredith Foster (Sanofi)	No disclosure on file
Susan Sparks	Susan Sparks has received personal compensation for serving as an employee of Sanofi. Susan Sparks has stock in Sanofi.
Benedikt Schoser	Benedikt Schoser has received personal compensation for serving as an employee of Alexion. Benedikt Schoser has received personal compensation for serving as an employee of Argenx. Benedikt Schoser has received personal compensation for serving as an employee of Amicus. Benedikt Schoser has received personal compensation for serving as an employee of Astellas. Benedikt Schoser has received personal compensation for serving as an employee of Denali. Benedikt Schoser has received personal compensation for serving as an employee of Avidity . Benedikt Schoser has received personal compensation for serving as an employee of Sanofi. Benedikt Schoser has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Taysha. Benedikt Schoser has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Astellas. Benedikt Schoser has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Encoded. Benedikt Schoser has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Kedrion.

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