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FDA Defers Action on Pompe Disease Candidate

FDA Defers Action on Pompe Disease Candidate

The FDA has issued a Deferred Action letter for Amicus Therapeutics’ biologic license application (BLA) for cipaglucosidase alfa, the biologic component of AT-GAA, its investigational dual therapy for muscle-wasting Pompe disease.

The agency said COVID-related travel restrictions prevented inspectors from visiting WuXi Biologics, the Chinese manufacturing plant that produces the enzyme, a recombinant form of the naturally occurring alpha-glucosidase (rhGAA) enzyme.

AT-GAA consists of a novel rhGAA for improved uptake and miglustat, an enzyme stabilizer.

The indefinite delay will be lifted only when FDA inspectors are able to visit the plant, the company said. But regulatory action on miglustat, the second component of the dual candidate is proceeding, even in the absence of a confirmed timeline.

Pompe disease

Pompe disease is a rare (estimated at 1 in every 40,000 births), inherited and often fatal disorder that disables the heart and skeletal muscles.  It is caused by mutations in a gene that makes an enzyme called acid alpha-glucosidase (GAA).  Normally, the body uses GAA to break down glycogen, a stored form of sugar used for energy. The enzyme performs its function in intracellular compartments called lysosomes.  Lysosomes are known to function as cellular clearinghouses; they ingest multiple substances including glycogen, which is converted by the GAA into glucose, a sugar that fuels muscles. In Pompe disease, mutations in the GAA gene reduce or completely eliminate this essential enzyme. 

November 3, 2022


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