Amicus Therapeutics Receives Positive CHMP Opinion for Pombiliti

Amicus Therapeutics

PHILADELPHIA, PAAmicus Therapeutics (Nasdaq: FOLD) announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) recently adopted a positive opinion recommending marketing authorization of cipaglucosidase alfa, a long-term enzyme replacement therapy (ERT) used in combination with miglustat for adults with late-onset Pompe disease (LOPD). A decision from the European Commission (EC) on cipaglucosidase alfa, the enzyme replacement therapy component of AT-GAA, is expected in the first quarter of 2023. Cipaglucosidase alfa will be commercialized under the brand name POMBILITI™. The Company expects a CHMP opinion of miglustat, the enzyme stabilizer component of AT-GAA, in the second quarter of 2023.

Late-onset Pompe disease is a rare, debilitating, and life-threatening lysosomal disorder caused by a deficiency of the enzyme acid alpha-glucosidase (GAA). Reduced or absent levels of GAA lead to the accumulation of the substrate glycogen in the lysosomes of muscles and other tissues. Disease severity ranges on a spectrum, but predominant manifestations are skeletal muscle weakness and progressive respiratory involvement.

“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. It is the realization of the work of so many individuals and teams dedicated to the mission of improving the lives of people living with Pompe disease,” said John F. Crowley, Executive Chairman and Founder of Amicus Therapeutics, Inc.

“Our team has worked tirelessly over the past decade to develop this innovative therapy, which we believe has the potential to address many of the unmet medical needs in this disease. We are grateful for the dedication and support from the Pompe community who have helped advance this therapy, especially the patients, families, and physicians who participated in our clinical studies. Based on the strength of the label and our launch readiness, once both components are approved, we believe there is significant commercial opportunity for AT-GAA in Europe and around the world,” said Bradley Campbell, President and Chief Executive Officer of Amicus Therapeutics, Inc.

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“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 Prof. Benedikt Schoser, Professor of Neurology at Ludwig-Maximillians-University of Munich LMU Department of Neurology. “The CHMP positive opinion and recommended indication reflect the robust data from AT-GAA’s clinical development program and gives me further hope for the potential of this innovative treatment alternative for people living with LOPD.”

AT-GAA is designed as a two-component therapy consisting of cipaglucosidase alfa, a long-term enzyme replacement therapy, administered in combination with miglustat, an oral enzyme stabilizer, for the treatment of adults with late-onset Pompe disease. Cipaglucosidase alfa is a recombinant human acid alpha-glucosidase enzyme (rhGAA) enriched with bis-mannose-6-phosphate designed to facilitate high-affinity uptake with retained capacity for processing into the most active form of the enzyme to break down glycogen.

The CHMP based its positive opinion on clinical data from the Phase 3 pivotal study (PROPEL), the only randomized, controlled trial in LOPD to include patients in the high unmet need ERT-experienced population, in addition to ERT-naïve patients. As anticipated and consistent with a recent opinion of another ERT in this disease space, the CHMP also determined cipaglucosidase alfa does not qualify as a New Active Substance (NAS).

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