Amicus plans migalastat filing in Japan in 2nd-qtr 2017

US rare and orphan disease focussed  biotech firm Amicus Therapeutics (Nasdaq: FOLD) plans to submit a Japanese New Drug Application (J-NDA) to request marketing authorization for migalastat, an oral precision medicine for Fabry disease, in the first half of 2017. The news moved Amicus shares up 1.2% to $6.59 by mid-morning New York trading.

Following a meeting with and written correspondence from the Pharmaceuticals and Medical Devices Agency (PMDA) in Japan, the J-NDA will be based on data from completed clinical studies with migalastat, including two pivotal Phase III studies, as well as a Phase I study that previously evaluated the pharmacokinetics (PK) of migalastat in Japanese volunteers. Taking into account data from Japanese patients included in the Phase III program and the similar PK properties in Japanese and non-Japanese individuals, the PMDA confirmed that these completed studies meet J-NDA submission requirements without the need to conduct an additional clinical study in Japan.

Japan Fabry ERT sales 13% of around $1.2 billion global market in 2015