Blog
RSS FeedPosted by Michael Wonder on 12 Feb 2018
U.S. FDA files new drug application under priority review for migalastat for treatment of Fabry disease
12 February 2018 - Six month PDUFA goal date is 13 August 2018. ...
Posted by Michael Wonder on 05 Feb 2018
Capricor receives FDA regenerative medicine advanced therapy designation for Duchenne muscular dystrophy therapy
5 February 2018 - CAP-1002 to benefit from expedited review program for drugs for unmet needs. ...
Posted by Michael Wonder on 04 Feb 2018
Alnylam announces FDA acceptance of new drug application and priority review status for patisiran, an investigational RNAi therapeutic for the treatment of hereditary ATTR amyloidosis
1 February 2018 - PDUFA date set for 11 August 2018. ...
Posted by Michael Wonder on 02 Feb 2018
Protalix BioTherapeutics’ pegunigalsidase alfa receives fast track designation from the U.S. FDA
31 January 2018 - Fast Track designation highlights high unmet medical need in the treatment of Fabry disease. ...
Posted by Michael Wonder on 28 Jan 2018
Medical miracles from FDA inefficiency
26 January 2018 - The economist behind the ‘priority review voucher,’ which advances treatments for neglected diseases. ...
Posted by Michael Wonder on 23 Jan 2018
FDA accepts investigational new drug application and grants fast track status for SOBI003 for the treatment of MPS IIIA
23 January 2018 - Swedish Orphan Biovitrum announces that the FDA has issued a study may proceed letter for the ...
Posted by Michael Wonder on 19 Jan 2018
Aquestive Therapeutics announces U.S. FDA filing acceptance of new drug application for clobazam oral soluble film to treat Lennox-Gastaut syndrome
18 January 2018 - Aquestive Therapeutics today announced that the U.S. FDA has accepted for review the company’s new drug application ...
Posted by Michael Wonder on 19 Jan 2018
Drug maker raises U.S. price of muscular dystrophy treatment
18 January 2018 - Cost of drug Emflaza, which came under scrutiny last year, will go up by 9% for regular ...
Posted by Michael Wonder on 23 Dec 2017
BioMarin receives anticipated notification of PDUFA extension for pegvaliase biologics license application to 28 May 2018
22 December 2017 - Regulatory review process proceeding in-line with company's expectations. ...
Posted by Michael Wonder on 19 Dec 2017
FDA approves novel gene therapy to treat patients with a rare form of inherited vision loss
19 December 2017 - Luxturna is the first gene therapy approved in the U.S. to target a disease caused by mutations ...
Posted by Michael Wonder on 19 Dec 2017
Republican bill to reduce tax credits for rare disease drugs
18 December 2017 - The target comes as a surprise under a Republican administration that has shown little interest in addressing ...
Posted by Michael Wonder on 17 Dec 2017
WTX101 granted fast track designation by the U.S. FDA for the treatment of Wilson disease
14 December 2017 - Wilson Therapeutics today announced that the U.S. FDA has granted WTX101 fast track designation for the treatment ...
Posted by Michael Wonder on 15 Dec 2017
Amicus Therapeutics submits new drug application to U.S. FDA for migalastat for treatment of Fabry Disease
14 December 2017 - Amicus Therapeutics submitted a new drug application to the U.S. FDA to request approval of the ...
Posted by Michael Wonder on 13 Dec 2017
Alnylam completes submission of new drug application to U.S. FDA for patisiran for the treatment of hereditary ATTR amyloidosis
12 December 2017 - Patisiran could become the first in a new class of medicines known as RNAi therapeutics. ...
Posted by Michael Wonder on 12 Dec 2017
FDA approves first drug for eosinophilic granulomatosis with polyangiitis, a rare disease formerly known as the Churg-Strauss Syndrome
12 December 2017 - The U.S. FDA today expanded the approved use of Nucala (mepolizumab) to treat adult patients with eosinophilic ...