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US FDA extends review of Amicus muscle disorder therapy for second time

The agency blamed the delay on pandemic-related travel restrictions that kept it from conducting a required manufacturing site inspection before the decision was due

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The US Food and Drug Administration (FDA) last week extended for the second time its review of Amicus Therapeutics’s experimental combination therapy to treat a muscle disorder called Pompe disease.

The agency blamed the delay on pandemic-related travel restrictions that kept it from conducting a required manufacturing site inspection before the decision was due.

Amicus had disclosed in a regulatory filing last month that an inspection of its partner WuXi Biologics’s manufacturing site in China had not been scheduled at the time.

Analysts from BTIG and Needham & Co said the extension will likely delay the launch of the therapy, AT-GAA, by a quarter.

Shares of the drugmaker were down three per cent at $10.1 in extended trading.

Amicus was seeking approval for the expanded use of its drug migulstat, in combination with its lab-made protein cipaglucosidase alfa, which together form AT-GAA.

The FDA had, in May, extended its review of both the components, saying it needed more time to go through the data submitted by the company.

Pompe disease is a rare genetic condition in which the body is unable to break down the complex sugar glycogen, leading to a buildup of the same, which causes muscular impairment. The condition may also lead to heart failure in young patients.

Amicus and partner Wuxi are the latest companies to have their review extended by the FDA over COVID-related travel curbs. China-based Beigene and Hutchmed, as well as the US-based Coherus BioSciences saw similar extensions earlier in the year.

Edits by EP News Bureau