European Commission approval for Pombiliti in patients with late-onset Pompe disease
Amicus Therapeutics, a patient-dedicated global biotechnology company focused on developing and commercializing novel medicines for rare diseases, announced that the European Commission (EC) has granted approval for Pombiliti (cipaglucosidase alfa), a long-term enzyme replacement therapy (ERT) used in combination with miglustat for adults with late-onset Pompe disease (LOPD)
The Company has submitted the previously requested analytical testing for miglustat, the enzyme stabilizer component of AT-GAA. The Committee for Medicinal Products for Human Use (CHMP) opinion for miglustat is expected in the second quarter of 2023.
The EC based its approval on clinical data from the Phase III 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. The EC approval of Pombiliti follows the positive opinion previously granted by the Committee for Medicinal Products for Human Use (CHMP).
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