Amicus knocked back by FDA in efforts to gain accelerated approval of AT-GAA in Pompe disease

Amicus Therapeutics disclosed Monday that the FDA determined that the current clinical package for AT-GAA is "not sufficient" to support accelerated approval for Pompe disease. The company said that it plans to generate further data to support a potential pathway for accelerated approval with in 2019, whilst starting a pivotal study this year. 

The feedback from the FDA, which came after a meeting with the regulator, follows similar recent guidance from the European Medicines Agency regarding a pathway for conditional marketing approval in the EU. AT-GAA consists of ATB200, a recombinant human acid alpha-glucosidase enzyme, to enhance uptake, co-administered with the pharmacological chaperone AT2221. 

Specifically, Amicus indicated that it will obtain data from 10 additional enzyme replacement therapy (ERT)-switch patients in a new cohort as part of an ongoing Phase I/II study, from which results are expected next year. The company also intends to provide clinical data through 18 months for the original 19 Phase I/II patients and complete a retrospective natural history study in about 100 ERT-treated Pompe disease patients, with data from both studies expected in the second half of this year. 

According to Amicus, the pivotal trial is expected to recruit around 100 patients with Pompe disease, including both ERT-switch patients and ERT treatment-naïve patients. The study's main goal will be six-minute walk with a primary treatment period of up to 12 months.

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