Blog
RSS FeedPosted 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. ...
Posted by Michael Wonder on 20 Sep 2016
Sarepta to charge $300K for Duchenne drug. ‘We tried to be reasonable,’ CEO says
19 September 2016 - The newly approved Sarepta Therapeutics drug for Duchenne muscular dystrophy will cost about $300,000 a year for ...
Posted by Michael Wonder on 20 Sep 2016
Did the FDA set ‘a dangerous precedent’ with its latest drug approval?
19 September 2016 - The experimental drug that the FDA approved Monday will only be used by a few thousand patients. ...
Posted by Michael Wonder on 20 Sep 2016
Behind the Sarepta drug approval was intense FDA bickering
19 September 2016 - The run-up to this week's approval of a Sarepta Therapeutics medicine to treat patients with Duchenne muscular ...
Posted by Michael Wonder on 19 Sep 2016
FDA grants accelerated approval to first drug for Duchenne muscular dystrophy
19 September 2016 - The U.S. FDA today approved Exondys 51 (eteplirsen) injection, the first drug approved to treat patients with ...
Posted by Michael Wonder on 11 Aug 2016
FDA accepts Marathon Pharmaceuticals’ new drug applications for deflazacort for the treatment of Duchenne muscular dystrophy and grants priority review
10 August 2016 - Deflazacort could be among the first FDA approved treatments for this devastating genetic disorder. ...
Posted by Michael Wonder on 11 Aug 2016
Heart of bureaucratic darkness
9 August 2016 - Why won’t the FDA make a decision on a muscular dystrophy drug? ...
Posted by Michael Wonder on 04 Aug 2016
More on FDA breakthrough therapies
4 August 2016 - The FDA has granted 148 breakthrough therapy designations as at 3 August 2016. Most of the ...