Amicus Says FDA Okays NDA Submission For Fabry Disease Treatment; Stock Up

(RTTNews) - Amicus Therapeutics (FOLD) said that it plans to submit a new drug application (NDA) to the U.S. FDA for the oral precision medicine migalastat for Fabry disease in the fourth quarter of 2017. Based on a series of discussions with and written communication received from the FDA, the Agency has informed Amicus that it may now submit an NDA for migalastat.

In the Tuesday's pre-market trade, FOLD is currently trading at $13.44, up $3.18 or 30.99%.

Amicus is preparing the NDA submission under Subpart H, which provides for accelerated approval. Amicus intends to base its NDA on existing data, including reduction in disease-causing substrate (GL-3), as well as the totality of data from completed clinical studies.

Progressive accumulation of GL-3 is believed to lead to the morbidity and mortality of Fabry disease, including pain, kidney failure, heart disease and stroke. An additional Phase 3 study previously requested by the Agency to assess Gastrointestinal (GI) symptoms is no longer required prior to an NDA submission.