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New Phase 3datapresented at WORLDSymposium™reinforce Nexviazyme®(avalglucosidasealfa) as potential new standard of care for all people living with late-onset Pompedisease
Paris, February 24, 2023. Today, at WORLDSymposiumTM, data from the Phase 3 COMET study long-term extension showed sustained treatment effect of Nexviazyme® (avalglucosidase alfa) over nearly three years in late-onset Pompe disease (LOPD) patients who were treatment-naïve as well as those who switched from long-time standard of care, alglucosidase alfa, during the 96-week extension period. Additionally, a separate analysis of respiratory function showed clinical benefit over two years for patients who switched to Nexviazyme regardless of prior response to alglucosidase alfa.
Priya S. Kishnani, MDC.L. and Su Chen Professor of Pediatrics; Medical Director, YT and Alice Chen Pediatrics Genetics and Genomics Center; and Division Chief, Medical Genetics, Duke University Medical Center“The resultspresented at the 2023 WORLDSymposiumcontinue to build on the datafrom the clinical trialson the value and long-term impact Nexviazymemayprovide for a wide variety of people living with late-onset Pompedisease, from newly diagnosed patients to those who have been previously treated with alglucosidasealfa. Further,when examining patients treated with Nexviazymewho switched from using alglucosidasealfa, these data show clinical benefitbothforpatientswho were stableonalglucosidasealfa andthose who experienced sub-optimal response or decline.”
Bill SiboldGlobal Head of Specialty Care, Sanofi“Theselong-termdataover nearly three yearsadd to the robust body of evidence that consistently shows theclinical benefit and durability of effectof treatmentwith Nexviazymeforboth treatment-experienced and treatment-naïve people living withlate-onset Pompedisease.Wecontinue to learn from our 20 years of experience in Pompedisease and build on our findings in an effort toadvancethe standard ofcare.”
Nexviazyme is a monotherapy approved in the US and other markets for the treatment of LOPD. It is also approved for infantile-onset Pompe disease (IOPD) in certain markets outside of the US.
Nexviazymemaintainedtreatment effect at 145 weeks
The Phase 3 COMET trial enrolled 100 previously untreated LOPD patients who were randomized to receive either Nexviazyme (20 mg/kg) or alglucosidase alfa (20 mg/kg) every two weeks for 49 weeks during the double-blind primary analysis period. During the open-label extension period, long-term efficacy and safety outcomes were assessed up to 145 weeks in patients who continued their treatment with Nexviazyme (n=51), as well as those who switched to treatment with Nexviazyme from alglucosidase alfa (n=44) at the conclusion of the primary analysis period (Week 49).
After nearly three years (145 weeks), changes from baseline (least squares mean [standard error]) showed:
The safety profile during treatment with Nexviazyme was comparable between both study groups (those who started Nexviazyme in the primary analysis period and those who switched to Nexviazyme during the extension period). No new safety signals were observed in patients who switched from alglucosidase alfa to Nexviazyme during the extension period. Across both Nexviazyme treatment groups, five patients discontinued treatment during the extension period due to six adverse events (AEs); four were treatment-related (ocular hyperemia, erythema, urticaria, respiratory distress), and two were non-treatment-related (acute myocardial infarction, pancreatic adenocarcinoma).
Clinical benefits seenover two years for patients who switched to Nexviazyme
Subgroup analyses were also performed to assess respiratory function outcomes for the 44 patients who switched to Nexviazyme in the long-term extension period of the Phase 3 COMET study. Patients were divided into two subgroups, responders (n=14) and non-responders (n=30) based on individual responses to initial treatment with alglucosidase alfa, as measured by the change in FVC percent-predicted (ΔFVC %predicted/year) pre- (baseline-week 49) and post-switch (week 49-week 145). Findings showed clinical benefits over two years following switch to Nexviazyme regardless of prior response to alglucosidase alfa:
