Avalglucosidase alfa-ngpt (Nexviazyme®) treatment via home infusion under healthcare professional supervision might be a safe option for patients with Pompe disease (PD), according to observations from the 3 clinical trials COMET (NCT02782741), NEO-EXT (NCT02032524), and Mini-COMET (NCT03019406).

The results of the new study published in Molecular Genetics and Metabolism will be presented at the 18th Annual WORLDSymposium, being held at the Manchester Grand Hyatt in San Diego, CA from February 7-11, 2022.

Six patients with late-onset Pompe disease (LOPD) experienced safety events. One of the patients, a 32-year-old female enrolled in COMET, had an infusion-associated reaction (IAR) during her first home infusion.

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“The patient experienced eyelid edema and flushing, both non-serious adverse events (AEs) of special interest and mild intensity IARs, 2 h after infusion initiation,” the authors said. The situation was managed by interrupting the infusion and administering dexchlorpheniramine, with the patient recovering the same day.

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After the reported event, the patient received 4 infusions at the study site, followed by 18 home infusions before data cut off. No additional IARs were reported in either of the settings.

The remaining 5 patients reported 10 nontreatment-related nonserious AEs. No safety events occurred in any of the patients with infantile-onset PD (IOPD). The study enrolled 16 patients diagnosed with PD who received avalglucosidase alfa-ngpt via home infusion (279 infusions in total) under healthcare professional supervision.

Avalglucosidase Alfa-ngpt for Late-Onset Pompe Disease Approved in Canada

Patients with LOPD received two 36 home infusions during the COMET trial (n=12) or 13 or 32 home infusions during the NEO-EXT trial (n=2). Two patients with IOPD received 6 or 8 home infusions during the Mini-COMET trial.


Díaz-Manera J, Hughes D, Béhin A, et al. Home-infusion experience in patients with Pompe disease receiving avalglucosidase alfa during three clinical trials (COMET, NEO-EXT, and Mini-COMET). Mol Genet Metab. 2022;135(2):S38. doi:10.1016/j.ymgme.2021.11.084