FDA hits Amicus with delay on 'breakthrough' Pompe drug
AT-GAA, Amicus Therapeutics’ lead Phase III drug for Pompe disease, was at the center of attention during the Q1 call with investors Monday. Execs talked about their high hopes for an approval by the July 29 PDUFA date, and analysts were asking questions about launch preparation.
The FDA, though, needs more time to make a decision.
Just after the market closed on Tuesday, Amicus put out word that the FDA has pushed back its review period by 90 days. The agency was dealing with two separate applications — a BLA for cipaglucosidase alfa and an NDA for miglustat — for the two components of AT-GAA, with PDUFA dates scheduled two months apart.
The delay marks another speed bump for Amicus, which was forced to call off a spinout of its gene therapy unit via a SPAC merger in the wake of a setback.
By combining a recombinant human acid α-glucosidase with an enzyme stabilizer, Amicus had hoped it could bring a next-gen approach to patients with Pompe disease. Although the drug, which was bestowed breakthrough therapy designation, failed to beat Lumizyme, Sanofi’s enzyme replacement therapy, in a Phase III trial, the biotech pushed ahead citing (statistically insignificant) improvements in favor of AT-GAA.
Amicus said the FDA is still reviewing newly submitted information as part of ongoing reviews, but that it is not related to requests for additional clinical data.
Besides, they noted, the additional time should allow for the agency to inspect the manufacturing sites of WuXi Biologics, its contract manufacturer, in China. Uncertainty around that inspection had been a key caveat for execs who were otherwise upbeat about an OK.
It continues to expect regulators to approve the applications together, the company added.
Social: John Crowley, Amicus CEO (via YouTube)