Blog
RSS FeedPosted 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 ...
Posted by Michael Wonder on 10 Sep 2017
Sobi receives approval from the FDA for once-daily dosing frequency of Orfadin for the treatment of HT-1
5 September 2017 - Sobi has received approval from the U.S. FDA for a reduced dosing frequency for Orfadin (nitisinone) ...
Posted by Michael Wonder on 07 Sep 2017
Global Blood Therapeutics receives rare paediatric disease designation from FDA for GBT440 for treatment of sickle cell disease
5 September 2017 - Global Blood Therapeutics today announced that the U.S. FDA has granted rare paediatric disease designation to GBT440 ...
Posted by Michael Wonder on 02 Aug 2017
FDA approves Kalydeco (ivacaftor) for more than 600 people ages 2 and older with cystic fibrosis who have certain residual function mutations
1 August 2017 - Vertex Pharmaceuticals today announced that the U.S. Food and Drug Administration (FDA) has approved Kalydeco (ivacaftor) ...
Posted by Michael Wonder on 03 Jul 2017
EMA and FDA encourage use of innovative approaches in the development of medicines for Gaucher disease
3 July 2017 - Approach aims to facilitate development of medicines for rare paediatric diseases in general. ...
Posted by Michael Wonder on 29 Jun 2017
FDA unveils plan to eliminate orphan designation backlog
29 June 2017 - Today, the U.S. Food and Drug Administration unveiled a strategic plan to eliminate the agency’s existing ...
Posted by Michael Wonder on 23 Jun 2017
Haegarda (C1 esterase inhibitor subcutaneous [human]) FDA approval
23 June 2017 - CSL Limited today announced that the U.S. FDA has approved CSL Behring’s Haegarda (C1 esterase inhibitor ...
Posted by Michael Wonder on 23 Jun 2017
FDA approves first subcutaneous C1 esterase Inhibitor to treat rare genetic disease
22 June 2017 - The U.S. FDA today approved Haegarda, the first C1 esterase inhibitor (human) for subcutaneous administration to ...