New data indicate that treatment with Nexviazyme® (avalglucosidase alfa) has significantly improved ptosis, or drooping eyelid, in pediatric patients with infantile-onset Pompe disease (IOPD) over nearly three years. These findings, presented at the 20th annual WORLDSymposiumTM, include positive safety results from the Phase 3 Baby-COMET trial, the first study of avalglucosidase alfa in treatment-naïve patients with IOPD in over 20 years. Ptosis affects around 50% of IOPD patients, potentially leading to vision loss and reduced quality of life. Nexviazyme is a monotherapy approved in the US and other markets for juvenile and adult patients with late-onset Pompe disease (LOPD). It is also approved for IOPD in certain markets outside of the US.
Dr. Priya S. Kishnani, C.L. and Su Chen Professor of Pediatrics at Duke University Medical Center, emphasized the significant improvements in ptosis seen in IOPD patients treated with avalglucosidase alfa.
Key findings surrounding avalglucosidase alfa use in late-onset Pompe disease (LOPD) were also presented at WORLDSymposiumTM 2024. This included positive long-term results from the NEO-EXT study up to eight years and real-world findings from the international Pompe Registry sponsored by Sanofi, showcasing patients who switched to avalglucosidase alfa from the long-time standard of care, alglucosidase alfa.
Dr. Alaa Hamed, Global Head of Medical Affairs, Rare Diseases at Sanofi, highlighted that the presented data build upon existing evidence supporting the value of avalglucosidase alfa in treating Pompe disease. The data demonstrate the therapy’s long-term durability, favorable efficacy, and safety profile across a wide range of patient types and clinical circumstances.
In the comparative COMET study, fewer LOPD patients in the avalglucosidase alfa group reported at least one infusion-associated reaction (IAR) compared to the alglucosidase alfa group. The most frequently reported treatment-emergent IARs in the avalglucosidase alfa group were pruritus and urticaria, with the majority being mild or moderate. Hypersensitivity reactions were observed in both groups, and the majority of these reactions were also mild or moderate.
The Phase 2 Mini-COMET study, evaluating the efficacy and safety of avalglucosidase alfa in patients under 18 years of age with IOPD, showed clinically meaningful improvements in ptosis over nearly three years. Safety results from the Phase 3 Baby-COMET trial, the first study evaluating the safety and efficacy of avalglucosidase alfa in treatment-naïve patients with IOPD, revealed no unexpected safety issues at the data cut-off.
Real-world evidence from the international Pompe Registry sponsored by Sanofi demonstrated that patients switching from long-time standard of care, alglucosidase alfa, to avalglucosidase alfa had stabilized respiratory function and mobility. Results from the NEO-EXT trial’s eight-year follow-up indicated a trend of stabilization over time in muscle quantitative magnetic resonance imaging (qMRI), supporting the clinical stability demonstrated in lung capacity and physical endurance.
