Amicus Therapeutics Publishes Positive PROPEL Study Data for Pompe Disease Treatment

Amicus Therapeutics Inc. (NASDAQ:FOLD) is one of the best biotech stocks to invest in now. On June 3, Amicus Therapeutics announced the publication of a post-hoc analysis of data from the ERT-experienced cohort of the PROPEL study in Muscle and Nerve. The analysis focused on the efficacy of Pombiliti (cipaglucosidase alfa-atga) + Opfolda (miglustat) (cipa+mig) in adults with late-onset Pompe disease/LOPD.

The results indicated that subjects who switched from alglucosidase alfa to cipa+mig generally achieved improvements or stability in most measured outcomes. The PROPEL study was a 52-week, double-blind, randomized global study that evaluated the efficacy, safety, and tolerability of cipa+mig against non-US-approved alglucosidase alfa + placebo.

Amicus Therapeutics Publishes Positive PROPEL Study Data for Pompe Disease Treatment

Doctors in a lab coat attending to a patient receiving enzyme replacement therapies.

Pombiliti + Opfolda is a two-component therapy. Pombiliti (cipaglucosidase alfa-atga) is a bis-M6P-enriched rhGAA designed for high-affinity uptake and efficient processing. Opfolda (miglustat) is an oral enzyme stabilizer that helps reduce the loss of enzyme activity in the blood. Late-onset Pompe disease is an inherited lysosomal disorder caused by a deficiency of the enzyme acid alpha-glucosidase/GA, leading to glycogen accumulation in cells. This progressive disease causes debilitating muscle weakness throughout the body, particularly affecting skeletal muscles and those involved in breathing.

Amicus Therapeutics Inc. (NASDAQ:FOLD) is a biotechnology company that discovers, develops, and delivers novel medicines for rare diseases in the US and internationally.

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Disclosure: None. This article is originally published at Insider Monkey.