The Committee for Medicinal Products for Human Use (CHMP) has receives positive feedback regarding the marketing authorization of cipaglucosidase alfa (Pombiliti), developed by Amicus Therapeutics as a treatment for late-onset Pompe disease, according to a recent press release.

Cipaglucosidase alfa is a key component of the AT-GAA therapy along miglustat. It acts as a long-term enzyme replacement therapy with a high-affinity uptake through the M6P receptor, while miglustat acts as an oral enzyme stabilizer. The 2-component therapy has been shown to improve respiratory and musculoskeletal measures in patients with late-onset Pompe disease.

“Today’s positive CHMP opinion for Pombiliti (cipaglucosidase alfa) is a significant milestone and major step towards bringing this much-needed new treatment for all adults living in the EU with late-onset Pompe disease,” said John F. Crowley, executive chairman and founder of Amicus Therapeutics, Inc.

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The positive CHMP opinion is based on the findings of phase 3 PROPEL pivotal trial, which compared the tolerability and efficacy of AT-GAA therapy with that of alglucosidase alfa. Results from the study showed a significant improvement in muscle strength, pulmonary function, and motor function in patients receiving AT-GAA therapy.

The trial was the first controlled randomized trial to include patients with late-onset Pompe disease within the enzyme replacement therapy-experienced population as well as patients who were enzyme replacement therapy naïve.

“This significant milestone moves AT-GAA closer to the LOPD community, where there is a high medical need for novel treatment options across patients, including those naïve and experienced to current treatments,” said Professor Benedikt Schoser, a professor of neurology at Ludwig-Maximillians-University of Munich LMU Department of Neurology.

The decision of the European Commission (EC) on cipaglucosidase alfa is still pending, and it is expected within the first trimester of 2023. Furthermore, Amicus Therapeutics expects a favorable opinion on miglustat by the second quarter of 2023. Cipaglucosidase alfa will be commercialized under the brand name POMBILITI™.


Amicus therapeutics receives positive CHMP opinion for Pombiliti™ (cipaglucosidase alfa) for late-onset Pompe Disease. News release. Amicus Therapeutics; December 16, 2022.