FDA Grants Breakthrough Therapy Designation to Amicus

On Feb 25, 2019

The U.S. Food and Drug Administration (FDA) granted Cranbury-based Amicus Therapeutics a Breakthrough Therapy Designation (“BTD”) to AT-GAA for the treatment of late onset Pompe disease. AT-GAA is the first investigational product for Pompe disease to receive BTD.

Breakthrough Therapy Designation was instituted by the FDA in 2012 to expedite the development and review of drugs that target serious conditions. To receive BTD there must be preliminary clinical evidence indicating that the drug may demonstrate substantial improvement on a clinically significant endpoint over available therapies.

John F. Crowley, chairman and chief executive officer of Amicus Therapeutics, stated, “There is an urgent need for new, second generation therapies for people living with lysosomal storage disorders, especially in a disease like Pompe. This important Breakthrough Therapy Designation from the FDA reflects the clinical data for our novel Pompe treatment paradigm AT-GAA. The BTD here also is based on the significant unmet need that remains for people living with Pompe disease, despite an approved therapy. We have been very pleased with the level of collaboration among the Amicus team, physicians, patients and the FDA in advancing AT-GAA. This BTD, together with our results from the Phase 2 study and ongoing PROPEL pivotal study, support our strategy to advance AT-GAA as quickly as possible with the potential to become the new standard of care for all persons living with Pompe disease.”

Amicus continues to expect that the pivotal PROPEL study (also referred to as ATB200-03) will be needed to support full approval of AT-GAA. Based on regulatory feedback from both the U.S. FDA and the European Medicines Agency (EMA), the company expects the AT-GAA development plan, including a planned pediatric study, to support a broad indication, for ERT-switch and treatment-naïve Pompe patients.

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