Alternative medicines

The Lancet Neurology Publishes Pivotal Phase 3 PROPEL Study Results of AT-GAA in Late-Onset … – The Bakersfield Californian

Summary

Peer Reviewed Results from PROPEL Show Treatment with AT-GAA Provided Clinically Meaningful Improvements Over Standard of Care, including ERT Experienced Patients with High Unmet Need

AT-GAA Deemed to Provide a Differentiated Mechanism of Action and Potential Alternative Treatment Option for People Living with Late-onset Pompe Disease

PHILADELPHIA, Nov. 18, 2021 (GLOBE NEWSWIRE) — Amicus Therapeutics (Nasdaq: FOLD), a patient-dedicated global biot…….

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Peer Reviewed Results from PROPEL Show Treatment with AT-GAA Provided Clinically Meaningful Improvements Over Standard of Care, including ERT Experienced Patients with High Unmet Need

AT-GAA Deemed to Provide a Differentiated Mechanism of Action and Potential Alternative Treatment Option for People Living with Late-onset Pompe Disease

PHILADELPHIA, Nov. 18, 2021 (GLOBE NEWSWIRE) — Amicus Therapeutics (Nasdaq: FOLD), a patient-dedicated global biotechnology company focused on developing and commercializing novel medicines for rare diseases, announced today that the data from the Phase 3 PROPEL pivotal trial, assessing the efficacy, safety and tolerability of AT-GAA in adults with late-onset Pompe disease compared to the standard of care, alglucosidase alfa, were published online in The Lancet Neurology. The manuscript includes data on the primary and key secondary endpoints, which were previously reported, as well as additional secondary endpoints. Based on the outcomes observed in the key domains of Pompe disease (muscle strength, pulmonary and motor function, patient-reported outcomes and biomarkers), the peer reviewed results determined the PROPEL data showed clinically meaningful improvements over standard of care, even among those who had been receiving approved therapy for at least 2 years, a subgroup that has been shown to plateau or decline after several years on treatment. AT-GAA is a two-component therapy consisting of cipaglucosidase alfa, an enhanced phosphorylated enzyme and miglustat, an enzyme stabilizer, thereby providing a different mechanism of action compared with alglucosidase alfa.

“Pompe disease is a rare genetic disease that causes premature death and has a debilitating effect on people’s lives. There is significant unmet medical need that persists within Pompe disease and patients are in need of new, effective and safe therapies. The outcomes of the PROPEL study demonstrate meaningful improvements in motor and respiratory functions in patients who are living with Pompe disease,” stated Prof. Benedikt Schoser, Professor of Neurology at Ludwig-Maximillians-University of Munich LMU Department of Neurology. “Results are particularly encouraging for the population switching from standard of care ERT to AT-GAA, who saw positive improvements in key manifestations of the disease.”

John F. Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics, stated, “We are pleased that The Lancet Neurology has published our PROPEL pivotal data in adults living with late-onset Pompe disease. Based on the clinical data we have seen from the Phase 3 trial, in addition to the body of data that we have accumulated over nearly a decade of preclinical and clinical studies, we have generated a compelling data set that supports the potential of AT-GAA becoming the next standard of care in the treatment of Pompe disease. We are honored by this publication and would like to thank all of the investigators, patients and families who participated in the study.”

The U.S. Food and Drug Administration (FDA) previously granted Breakthrough Therapy designation for AT-GAA and accepted for review the Biologics License Application (BLA) and the New Drug Application (NDA). The FDA has set a Prescription Drug User Fee Act action date of May 29, 2022 for the NDA and July 29, 2022 for the BLA. In …….

Source: https://www.bakersfield.com/ap/news/the-lancet-neurology-publishes-pivotal-phase-3-propel-study-results-of-at-gaa-in-late/article_44c59b11-ce4f-5b94-8615-e228ad9fc06a.html