Expert Interview
Discussing Amicus’ AT-GAA (cipaglucosidase alfa) for Pompe Disease, following PDUFA Date Extensions
Ticker(s): FOLDA geneticist with experience treating Pompe Disease.
Please describe your clinical practice. How many patients with Pompe Disease do you see on a yearly basis? What is the standard of care, and how well do patients respond to it?
Could you please discuss the Reduction in Biomarkers of muscle damage and disease substrate and the Effect on Muscle Tissue, judging by the results of the six-minute walk test distance?
- Functional Outcomes (n=23):Motor function: Ambulatory cohorts showed durable mean improvements from baseline that were sustained for up to 36 months of follow-up.Amongst ERT-naïve patients, 6MWT distance increased in 6/6, 5/5, and 4/5 patients at months 12, 24, and 36, respectively. The ERT-naïve patients showed mean increases of 57 meters at month 12 (n=6), 61 meters at month 24 (n=5), and 44 meters at month 36 (n=5).6MWT distance increased in 13/16, 8/10, and 6/8 ERT-experienced patients at months 12, 24, and 36, respectively. The ERT-experienced patients showed mean increases of 34 meters at month 12 (n=16), 21 meters at month 24 (n=10), and 48 meters at month 36 (n=8).Muscle Strength: Ambulatory and non-ambulatory patients, including ERT-experienced and ERT-naïve, showed improvements in strength testing as assessed by manual muscle testing (MMT) and improvements were maintained out to 36 months.Pulmonary Function: Pulmonary function improved in ERT-naïve patients and was generally stable in ERT-experienced patients.In ERT-naïve patients, mean change in percent predicted forced vital capacity (FVC), one of the main measures of pulmonary function in Pompe disease, was +4.5% at month 12 (n=6), +6.8% at month 24 (n=5), and +6.2% at month 36 (n=5).In ERT-experienced patients, mean change in % predicted FVC was -1.3% at month 12 (n=16), -0.9% at month 24 (n=10), and -0.4% at month 36 (n=8).
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How does AT-GAA compare with standard of care in reducing creatine kinase and disease substrate (urine hexose tetrasaccharide or Hex4) ? How critical is persistence and durability ?
What are some of the patients’ still unmet needs, and how do you think those could be addressed in the future?
What is the likelihood of AT-GAA to become the next standard of care in Pompe disease?
How likely would you be to switch patients from standard of care ERT to AT-GAA?
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