20:35 , Aug 10, 2018 |  BC Extra  |  Company News

Amicus' Galafold wins accelerated approval from FDA

FDA granted accelerated approval to Galafold migalastat from Amicus Therapeutics Inc. (NASDAQ:FOLD) to treat Fabry disease in adults who have amenable mutations. Amicus said the U.S. approval is the first for a new Fabry disease...
13:04 , Feb 16, 2018 |  BC Week In Review  |  Clinical News

Priority Review for Amicus' migalastat

FDA accepted and granted Priority Review to an NDA from Amicus Therapeutics Inc. (NASDAQ:FOLD) for migalastat (AT1001) to treat Fabry's disease in patients 16 years and older who have amenable mutations. The PDUFA date is...
22:06 , Feb 12, 2018 |  BC Extra  |  Company News

Priority Review for Amicus' migalastat

FDA accepted and granted Priority Review to an NDA from Amicus Therapeutics Inc. (NASDAQ:FOLD) for migalastat (AT1001) to treat Fabry's disease in patients 16 years and older who have amenable mutations. The PDUFA date is...
19:47 , Dec 15, 2017 |  BC Week In Review  |  Clinical News

Amicus submits NDA for Fabry candidate

Amicus Therapeutics Inc. (NASDAQ:FOLD) submitted an NDA to FDA for migalastat (AT1001) to treat patients 16 years and older with Fabry disease who have amenable mutations. In July, Amicus said FDA would no longer require...
19:31 , Jul 14, 2017 |  BC Week In Review  |  Clinical News

FDA clears Amicus to submit migalastat NDA without new trial

Rare disease company Amicus Therapeutics Inc. (NASDAQ:FOLD) said FDA would no longer require an additional Phase III study of migalastat (AT1001) to treat Fabry's disease, clearing the way for the company to submit an NDA...
00:08 , Jul 12, 2017 |  BC Extra  |  Company News

FDA clears Amicus to submit migalastat NDA without new trial

Rare disease company Amicus Therapeutics Inc. (NASDAQ:FOLD) gained $2.66 (26%) to $12.92 on Tuesday after it said FDA would no longer require an additional Phase III study of migalastat (AT1001) to treat Fabry's disease, clearing...
01:47 , Mar 4, 2017 |  BioCentury  |  Politics, Policy & Law

Seeing is believing

Pompe’s disease patient Megan Crowley and her father, biotech CEO John Crowley, accepted an invitation to meet with Donald Trump and attend his speech on Tuesday to present the hopes and needs of the rare...
22:38 , Jan 20, 2017 |  BioCentury  |  Emerging Company Profile

Aiming for durability

Avrobio Inc. is developing cell therapies that are transduced with lentivirus ex vivo to express therapeutic proteins. The approach could provide long-lasting treatments for patients with monogenic rare diseases or cancer. Data from Phase I...
00:40 , Nov 29, 2016 |  BC Extra  |  Company News

Amicus again slows migalastat's NDA timeline

Amicus Therapeutics Inc. (NASDAQ:FOLD) shed $2.29 (28%) to $6.03 in early after-hours trading Monday after it revealed a new timeline for its planned NDA submission for migalastat (AT1001) to treat Fabry's disease. The company now...
08:00 , Mar 7, 2016 |  BC Week In Review  |  Clinical News

Migalastat: Additional Phase III data

Data from paired biopsies of 8 treatment-naive Fabry’s disease patients with amenable genetic mutations in the double-blind, placebo-controlled, international Phase III FACETS (Study 011) trial showed that 150 mg oral migalastat every other day significantly...