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RSS FeedPosted 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 ...
Posted by Michael Wonder on 29 Oct 2017
Atara Biotherapeutics receives rare paediatric disease designation from FDA for ATA230 for treatment of congenital cytomegalovirus infection
26 October 2017 - Atara Biotherapeutics today announced that ATA230 was granted rare paediatric disease designation for the treatment of ...
Posted by Michael Wonder on 26 Oct 2017
PTC Therapeutics receives complete response letter for ataluren's NDA
25 October 2017 - PTC Therapeutics today announced that the Office of Drug Evaluation I of the U.S. FDA has ...
Posted by Michael Wonder on 10 Oct 2017
The lifecycle of a rare disease cancer drug
10 October 2017 - One pharma company’s medicine illustrates how firms can benefit from different layers of market-exclusivity protections. ...
Posted by Michael Wonder on 07 Oct 2017
FDA awards six grants for natural history studies in rare diseases
6 October 2017 - The U.S. FDA today announced it has awarded six new research grants for natural history studies ...
Posted by Michael Wonder on 07 Oct 2017
FDA awards 15 grants for clinical trials to stimulate product development for rare diseases
6 October 2017 - The U.S. FDA today announced that it has awarded 15 new clinical trial research grants totalling ...
Posted by Michael Wonder on 25 Sep 2017
U.S. FDA grants fast track designation for Amicus Therapeutics' migalastat for treatment of Fabry disease
19 September 2017 - New drug application submission on track for 4Q17. ...
Posted by Michael Wonder on 13 Sep 2017
FDA’s Gottlieb plans to close an orphan drug loophole
12 September 2017 - Amid rising complaints that drug makers are exploiting loopholes to win approval of so-called orphan drugs, ...
Posted by Michael Wonder on 12 Sep 2017
FDA is advancing the goals of the Orphan Drug Act
12 September 2017 - Three months ago, I committed to fully eliminate a backlog of about 200 orphan drug designation requests ...