BC Extra | Aug 12, 2019
Politics & Policy

Aug. 12 P&P Quick Takes: China to implement ethics committee; plus Senators call for action on AveXis and FDA’s Fabry guidance

China planning national science, technology ethics committee  China’s central committee for deepening overall reform approved a plan to establish a national science and technology ethics committee. The government said the committee is meant to improve...
BC Extra | Jul 16, 2019
Clinical News

July 15 Clinical Quick Takes: Zejula meets in Phase III for 1L ovarian cancer; plus Capricor, Avrobio and NIH

Zejula improves 1L ovarian cancer PFS  Zejula niraparib met the primary endpoint in the Phase III PRIMA trial to treat first-line ovarian cancer following platinum chemotherapy, significantly improving progression-free survival regardless of patients' biomarker status...
BC Extra | Aug 10, 2018
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...
BC Week In Review | Feb 16, 2018
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...
BC Extra | Feb 12, 2018
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...
BC Week In Review | Dec 15, 2017
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...
BC Week In Review | Jul 14, 2017
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...
BC Extra | Jul 12, 2017
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...
BioCentury | Mar 4, 2017
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...
BioCentury | Jan 20, 2017
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...
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