Sanofi Presents New Data on Nexviazyme for Pompe Disease
Sanofi has released new clinical data demonstrating the safety, benefit, and durability of Nexviazyme (avalglucosidase alfa) across various patient groups with Pompe disease, including pediatric and adult populations.
Sanofi announced new clinical data for Nexviazyme (avalglucosidase alfa), a treatment for Pompe disease. The findings, presented at the 20th annual WORLD Symposium, highlight the drug's clinical safety, benefits, and long-term durability in diverse patient populations.
Data indicate that Nexviazyme meaningfully improved ptosis, or drooping eyelid, in pediatric patients with infantile-onset Pompe disease (IOPD). These results stem from the long-term extension of the Phase 2 Mini-COMET study. Additionally, initial data from the Phase 3 Baby-COMET trial, evaluating avalglucosidase alfa in never-before-treated IOPD patients, were presented. Ptosis can lead to vision impairment and reduced quality of life, affecting approximately 50% of IOPD patients.
The symposium also featured presentations on Nexviazyme's use in late-onset Pompe disease (LOPD). Long-term results from the NEO-EXT study, extending up to eight years, along with real-world findings from the international Pompe Registry, support the drug's sustained efficacy and favorable safety profile.
Sanofi emphasized that the presented data reinforce the value of avalglucosidase alfa in treating Pompe disease. The company stated that the evidence showcases the therapy's durability and favorable efficacy and safety profile across a wide range of patient types and clinical circumstances. Researchers continue to follow scientific outcomes to address unmet needs.
In the comparative COMET study, fewer treatment-emergent infusion-related reactions (IARs) were reported in the avalglucosidase alfa group compared to the alglucosidase alfa group. The most frequent adverse events included pruritus and urticaria, with the majority of reactions being mild to moderate.