Sanofi’s enzyme replacement therapy (ERT) Nexviazyme (avalglucosidase alfa) has received US Food and Drug Administration (FDA) approval for the treatment of late-onset Pompe disease (LOPD), the French pharma company announced last week.

Nexviazyme is designed to target the mannose-6-phosphate (M6P) receptor – a key pathway for cellular uptake of ERT in Pompe disease.

In the Phase III COMET trial, comparing Nexviazyme to the older ERT drug Myozyme (alglucosidase alfa) in LOPD, patients treated with Nexviazyme had a 2.9-point improvement in forced vital capacity (FVC) – the study’s primary endpoint.

Also, when compared to baseline, patients treated with Nexviazyme walked 32.2 metres farther at week 49 and walked 30 metres farther than patient treated with Myozyme, at the same time point.

Serious adverse reactions were reported in two patients treated with Nexviazyme nad in three patients treated with Myozyme during the 49 week study period.

“Pompe disease is a debilitating and progressive condition that significantly inhibits mobility and breathing,” said Bill Sibold, executive vice president of Sanofi Genzyme.

“For decades, we’ve made it our responsibility to research how to target the M6P receptor, the key pathway for cellular uptake of enzyme replacement therapy. Nexviazyme is a potential new standard of care for people living with late-onset Pompe disease and delivers on our promise to pursue medicines for patients living with rare diseases,” he added.

Pompe disease is caused by a genetic deficiency or dysfunction of the lysosomal enzyme acid alpha-glucosidase (GAA), leading to a build-up of complex sugars in muscle cells throughout the body.

This in turn leads to irreversible damage to the muscles, including the diaphragm that support respiratory function as well as skeletal muscles which affects mobility, functional endurance and breathing.

LOPD – a form of Pompe disease which progressively damages muscles over time – can present at any age.

However, it can take seven to nine years before patients receive an accurate LOPD diagnosis, as the condition has a wide range of clinical presentations and because of its progressive nature.

US approval for Sanofi’s late-onset Pompe disease med Nexviazyme