Blog
RSS FeedPosted 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 ...
Posted by Michael Wonder on 17 Jan 2017
Drug makers manipulate orphan drug rules to create prized monopolies
17 January 2017 - More than 30 years ago, Congress overwhelmingly passed a landmark health bill aimed at motivating pharmaceutical ...
Posted by Michael Wonder on 31 Dec 2016
Costly drug for fatal muscular disease wins FDA approval
30 December 2016 - The FDA has approved the first drug to treat patients with spinal muscular atrophy, a savage ...
Posted by Michael Wonder on 29 Nov 2016
Amicus Therapeutics announces U.S. regulatory pathway for migalastat hydrochloride for Fabry disease
28 November 2016 - Regulatory plan for full approval pathway based on generation of additional gastro-intestinal symptoms data. ...
Posted by Michael Wonder on 21 Oct 2016
FDA official warns other drug makers not to copy Sarepta
20 October 2016 - Any company that plans to mimic the approach taken by Sarepta Therapeutics to win regulatory approval ...
Posted by Michael Wonder on 17 Oct 2016
FDA awards 21 grants to stimulate product development for rare diseases
17 October 2016 - The U.S. FDA today announced that it has awarded 21 new clinical trial research grants totaling ...
Posted by Michael Wonder on 16 Oct 2016
Bad medicine
15 October 2016 - Approving an unproven drug sets a worrying precedent. ...
Posted by Michael Wonder on 09 Oct 2016
Muscular dystrophy drug looks set for commercial success despite clinical doubts
3 October 2016 - A newly approved drug to treat Duchenne muscular dystrophy seems likely to be used by most ...
Posted by Michael Wonder on 03 Oct 2016
A golden ticket that fast tracks a medicine through the FDA
29 September 2016 - Sarepta Therapeutics won a big victory when its $300,000 muscular dystrophy drug was recently approved, but the ...
Posted by Michael Wonder on 23 Sep 2016
FDA approves expanded indications for Ilaris for three rare diseases
23 September 2016 - The U.S. FDA today approved three new indications for Ilaris (canakinumab). ...
Posted by Michael Wonder on 22 Sep 2016
The FDA did the right thing by approving a novel drug for a disabling disease
22 September 2016 - The FDA’s leadership did the right thing, according to Elaine Schattner in Forbes magazine. ...
Posted by Michael Wonder on 22 Sep 2016
FDA commissioner calls for Sarepta clinical trial to be retracted
22 September 2016 - In an unusual development, the US FDA Commissioner Dr. Robert Califf has indicated that a clinical trial ...
Posted by Michael Wonder on 20 Sep 2016
The boys who beat the FDA
19 September 2016 - The agency approves a new medicine after an ugly bureaucratic brawl. ...