EC approval for Amicus' Pombiliti
US rare diseases focused biotech Amicus Therapeutics (Nasdaq: FOLD) today 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).
Amicus says it 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.
In the USA, last October the US Food and Drug Administration (FDA) deferred action on the Biologics License Application (BLA) for cipaglucosidase alfa. This was due to restrictions on travel related to COVID-19, meaning the FDA was unable to conduct the required inspection of the WuXi Biologics manufacturing site in China during the review cycle.
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