Amicus Therapeutics Announces Regulatory and Clinical Updates for AT-GAA in Pompe Disease

- September 10th, 2018

Amicus Therapeutics (Nasdaq:FOLD), a global biotechnology company focused on discovering, developing and delivering novel medicines for rare metabolic diseases, announced today regulatory and clinical advancements in its development program AT-GAA for Pompe disease. As quoted in the press release: During the third quarter, Amicus held a Type C meeting with the United States Food and … Continued

Amicus Therapeutics (Nasdaq:FOLD), a global biotechnology company focused on discovering, developing and delivering novel medicines for rare metabolic diseases, announced today regulatory and clinical advancements in its development program AT-GAA for Pompe disease.

As quoted in the press release:

During the third quarter, Amicus held a Type C meeting with the United States Food and Drug Administration (FDA) in order to discuss the regulatory path for AT-GAA. Specifically, Amicus sought input on the design of a pivotal study for full approval for AT-GAA, other supplemental clinical studies in Pompe disease patients, and whether Amicus may pursue an Accelerated Approval pathway in the United States at this time. Amicus has now received final written minutes from the Type C meeting, in which the FDA noted “the importance of expediting new treatments to Pompe patients as fast as possible.”  Amicus has incorporated key elements of feedback from the FDA, including the Type C meeting, along with the prior scientific advice received from the European Medicines Agency (EMA) and plans to initiate a pivotal study in 2H18.

Click here to read the full press release.

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