Blog
RSS FeedPosted 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 ...
Posted by Michael Wonder on 18 Jun 2017
Fibrocell receives rare paediatric disease designation from FDA for FCX-013 for treatment of localised scleroderma
12 June 2017 - Gene therapy candidate under control of RheoSwitch therapeutic system technology is potential first-in-class treatment for chronic ...
Posted by Michael Wonder on 20 May 2017
How did Vertex win an FDA approval without running a clinical trial?
19 May 2017 - Vertex Pharmaceuticals scored a minor coup this week when the Food and Drug Administration signed off ...
Posted by Michael Wonder on 17 May 2017
FDA expands approved use of Kalydeco to treat additional mutations of cystic fibrosis
17 May 2017 - The U.S. FDA today expanded the approved use of Kalydeco (ivacaftor) for treating cystic fibrosis. ...
Posted by Michael Wonder on 16 May 2017
Imara receives rare paediatric disease designation from FDA for lead product candidate IMR-687 for sickle cell disease
15 May 2017 - Imara announced today that the U.S. FDA has granted rare paediatric disease designation to IMR-687, the ...
Posted by Michael Wonder on 02 May 2017
Horizon Pharma announces FDA approval to expand the age range for Ravicti (glycerol phenylbutyrate) oral liquid to people with urea cycle disorders two months of age and older
1 May 2017 - Horizon Pharma today announced the U.S. FDA has approved its supplemental new drug application to expand the ...
Posted by Michael Wonder on 27 Apr 2017
FDA approves first treatment for a form of Batten disease
27 April 2017 - The U.S. FDA today approved Brineura (cerliponase alfa) as a treatment for a specific form of Batten ...
Posted by Michael Wonder on 23 Mar 2017
An FDA program incentivising rare disease drugs will be investigated for abuses
23 March 2017 - In late 1982, Congress overwhelmingly passed the Orphan Drug Act, which was then signed into law ...
Posted by Michael Wonder on 13 Mar 2017
Rare diseases patients need more than miracles and Trumpcare
11 March 2017 - Never one to miss an opportunity, Trump cheapened Rare Disease Week by exploiting a young woman, Megan ...
Posted by Michael Wonder on 09 Mar 2017
PTC Therapeutics announces FDA acknowledgment of new drug application filing for Translarna for the treatment of nonsense mutation Duchenne muscular dystrophy
6 March 2017 - FDA has assigned a PDUFA date of 24 October 2017. ...
Posted by Michael Wonder on 13 Feb 2017
Grassley launches inquiry into orphan drugs after KHN investigation
10 February 2017 - Republican Sen. Chuck Grassley, chairman of the Senate Judiciary Committee, has opened an inquiry into potential ...
Posted by Michael Wonder on 09 Feb 2017
FDA approves drug to treat Duchenne muscular dystrophy
9 February 2017 - The U.S. FDA today approved Emflaza (deflazacort) tablets and oral suspension to treat patients age 5 ...