Blog
RSS FeedPosted 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 ...
Posted by Michael Wonder on 03 Dec 2017
ArmaGen’s AGT-181 granted fast track designation for the treatment of Hurler syndrome
30 November 2017 - ArmaGen today reported that the U.S. FDA has granted fast track designation to AGT-181, a novel, ...
Posted by Michael Wonder on 21 Nov 2017
FDA grants breakthrough therapy designation and orphan drug designation to PellePharm for topical patidegib in Gorlin syndrome
20 November 2017 - PellePharm today announced that the U.S. FDA has granted both breakthrough therapy designation and orphan drug ...
Posted by Michael Wonder on 17 Nov 2017
Alnylam initiates rolling submission of new drug application to U.S. FDA for patisiran for the treatment of hereditary ATTR amyloidosis
16 November 2017 - Company plans to complete submission by year end. ...
Posted by Michael Wonder on 16 Nov 2017
Akcea and Ionis announce acceptance of marketing applications in US, EU and Canada for volanesorsen for the treatment of FCS
15 November 2017 - FDA Prescription Drug User Fee Act goal date set for 30 August 2018. ...
Posted by Michael Wonder on 15 Nov 2017
FDA approves treatment for rare genetic enzyme disorder
15 November 2017 - The U.S. FDA today approved Mepsevii (vestronidase alfa-vjbk) to treat pediatric and adult patients with an ...
Posted by Michael Wonder on 06 Nov 2017
FDA approves first treatment for certain patients with Erdheim-Chester Disease, a rare blood cancer
6 November 2017 - FDA expands approval of Zelboraf (vemurafenib) to treat certain adult patients with Erdheim-Chester Disease, a rare cancer ...
Posted by Michael Wonder on 02 Nov 2017
FDA grants rare paediatric disease designation to ArQule's miransertib (ARQ 092) for the treatment of Proteus syndrome
1 November 2017 - ArQule today announced that the U.S. FDA has granted rare paediatric disease designation to miransertib (ARQ ...