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Abstract: FR-PO339

Baseline Characteristics of Patients With Fabry Disease Enrolled in the Pegunigalsidase Alfa Expanded-Access Program

Session Information

Category: Genetic Diseases of the Kidneys

  • 1102 Genetic Diseases of the Kidneys: Non-Cystic

Authors

  • Mehta, Ankit, Baylor University Medical Center at Dallas, Dallas, Texas, United States
  • Wilcox, William, Emory University School of Medicine, Atlanta, Georgia, United States
  • Nedd, Khan J., Infusion Associates, Grand Rapids, Michigan, United States
  • Bernat, John A., University of Iowa Hospital and Clinics Iowa City, Iowa City, Iowa, United States
  • Knoll, Jasmine, Phoenix Children’s Hospital, Phoenix, Arizona, United States
  • Jefferies, John L., The University of Tennessee Health Science Center, Memphis, Tennessee, United States
  • Longo, Nicola, University of Utah, Salt Lake City, Utah, United States
  • Wallace, Eric L., University of Alabama at Birmingham, Birmingham, Alabama, United States
  • Almon, Einat, Protalix Biotherapeutics, Carmiel, Northern, Israel
  • Alon, Sari, Protalix Biotherapeutics, Carmiel, Northern, Israel
  • Chertkoff, Raul, Protalix Biotherapeutics, Carmiel, Northern, Israel
  • Sullivan, Dawn, Chiesi USA, Inc, Boston, Massachusetts, United States
  • Rocco, Rossana, Chiesi USA, Inc, Boston, Massachusetts, United States
  • Koulinska, Irene, Chiesi USA, Inc, Boston, Massachusetts, United States
  • Goker-Alpan, Ozlem, Lysosomal & Rare Disorders Research & Treatment Center, Fairfax, Virginia, United States
Background

Fabry disease (FD) is a rare genetic disorder caused by deficiency in lysosomal enzyme, alpha-galactosidase A (α-Gal A), activity. Pegunigalsidase alfa (PA) is a novel PEGylated α-Gal A enzyme replacement therapy in development for FD. An expanded-access program (EAP) is offered in the US to adults with FD that cannot be adequately managed with approved drugs and who are not eligible for clinical trials. Characterizing patients in an EAP is critical to understanding unmet needs.

Methods

We collated baseline characteristics for patients with FD enrolled in the ongoing EAP in the US as of January 1, 2022.

Results

At the data cutoff date, 30 patients had enrolled in the EAP; primarily due to worsening disease symptoms and/or poor tolerability of infusions. There was a mean of 12.9 (SD 11.8) years since diagnosis and most patients had received agalsidase beta treatment (mean duration 7.1 [SD 4.7] years). Median (range) baseline eGFR was 91.4 mL/min/1.73m2 (14.1–131.9) and median concentration (range) of plasma globotriaosylsphingosine was 18.1 nM (0.8–134.6)

Conclusion

Assessment of patients in the PA EAP demonstrated gaps in current treatment options for FD. Some patients had experienced persistent symptoms despite treatment and/or poor tolerability of infusions and may benefit from the PA EAP. Follow-up of clinical outcomes from this EAP will be monitored and presented as data become available.

Funding

  • Commercial Support