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Amicus' rare disease drug denied accelerated approval pathway by US FDA

Amicus Therapeutics Inc. said the U.S. Food and Drug Administration denied an accelerated approval pathway for its rare disease drug candidate AT-GAA.

AT-GAA is being developed to treat Pompe Disease, a rare and inherited disorder caused by the deficiency of the enzyme acid alpha-glucosidase. The enzyme's deficiency results in the accumulation of glycogen, the stored form of glucose, in cells, leading to severe muscle weakness.

The U.S. FDA's accelerated approval pathway allows faster approval of drugs for serious conditions that fill an unmet medical need based on an initial clinical benefit.

According to the regulator, the current clinical data submitted by Amicus Therapeutics is not enough to be granted the accelerated approval pathway.

The rare-disease-focused company said it will continue to generate more data, including that from an ongoing phase 1/2 study as well as a natural history study in about 100 Pompe patients treated with an enzyme replacement therapy, to support further discussions on a potential pathway for accelerated approval with the FDA in 2019.

A natural history study follows a group of people over time who have or who are at risk of developing a disease, in order to collect health information and understand the disease.

The Cranbury, N.J.-based company said the data from these studies is expected to be available in the second half of 2018 or in 2019.

Amicus Therapeutics also plans to launch a pivotal study in the second half of 2018 for the full approval of AT-GAA.