An Open-Label Extension Study to Evaluate the Long Term Safety and Efficacy of Migalastat Hydrochloride Monotherapy in Subjects With Fabry Disease
Active, no longer recruiting
Phase of Trial: Phase III
Latest Information Update: 11 Jul 2017
At a glance
- Drugs Migalastat (Primary)
- Indications Fabry's disease
- Focus Adverse reactions; Registrational
- Sponsors Amicus Therapeutics
- 11 Jul 2017 According to an Amicus Therapeutics media release, the company 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.
- 11 Jul 2017 According to an Amicus Therapeutics media release, the company plans to submit a new drug application (NDA) to the U.S. FDA for 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.
- 03 Aug 2016 According to an Amicus Therapeutics media release, the company plans to submit a Japanese New Drug Application (J-NDA) for migalastat for the treatment of Fabry's disease in the first half of 2017. The application will include data from phase III pivotal studies (FACETS and ATTRACT) and long-term extension studies.