Blog
RSS FeedPosted by Michael Wonder on 08 Oct 2019
FDA awards 12 grants to fund new clinical trials to advance the development of medical products for the treatment of rare diseases
8 October 2019 - The U.S. FDA today announced that it has awarded 12 new clinical trial research grants totaling ...
Posted by Michael Wonder on 03 Sep 2019
The FDA and Sarepta: a window into the real world of drug regulation
3 September 2019 - It is hard to discern the true state of drug regulation from the outside, but two ...
Posted by Michael Wonder on 28 Aug 2019
European Commission approves Soliris (eculizumab) for the treatment of adults with neuromyelitis optica spectrum disorder
27 August 2019 - 98% of adult anti-aquaporin-4 antibody-positive patients treated with Soliris were relapse free compared to 63% receiving placebo ...
Posted by Michael Wonder on 20 Aug 2019
FDA accepts new drug application for VX-445 (elexacaftor), tezacaftor and ivacaftor combination treatment
20 August 2019 - FDA grants priority review of the application and sets a PDUFA target action date of 19 ...
Posted by Michael Wonder on 24 Jul 2019
Aeglea BioTherapeutics receives FDA breakthrough therapy designation for pegzilarginase for treatment of arginase 1 deficiency
24 July 2019 - Designation follows recently reported ARG1-D Phase 1/2 data demonstrating clinical response in patients rreated with pegzilarginase. ...
Posted by Michael Wonder on 22 Jul 2019
Vertex submits new drug application to the U.S. FDA for triple combination regimen of VX-445 (elexacaftor), tezacaftor and ivacaftor in cystic fibrosis
22 July 2019 - Application supported by positive results from two global Phase 3 studies in people with CF ages 12 ...
Posted by Michael Wonder on 11 Jul 2019
Reforming the Orphan Drug Act for the 21st century
11 July 2019 - The pharmaceutical market has undergone radical changes, including markedly increased prices for rare-disease drugs. ...
Posted by Michael Wonder on 04 Jul 2019
Ultomiris (ravulizumab) receives marketing authorisation from European Commission for adults with paroxysmal nocturnal hemoglobinuria
3 July 2019 - Ultomiris is the first approved, long-acting complement inhibitor for PNH, administered every other month, reducing the ...
Posted by Michael Wonder on 20 Jun 2019
U.S. FDA accepts supplemental biologics license application for Ultomiris (ravulizumab-cwvz) under priority review for the treatment of atypical haemolytic uremic syndrome
20 June 2019 - FDA sets target action date of 19 October 2019. ...
Posted by Michael Wonder on 19 Jun 2019
Abeona Therapeutics receives FDA fast track designation for ABO-202 AAV9 gene therapy in CLN1 disease
18 June 2019 - Abeona Therapeutics today announced that the U.S. Food and FDA has granted fast track designation to its ...
Posted by Michael Wonder on 11 Jun 2019
Jacobus prices its rare disease drug at half of what Catalyst charges, but will doctors prescribe it?
10 June 2019 - After weeks of anticipation, Jacobus Pharmaceutical, a small, family-run drug maker, has priced its rare disease ...
Posted by Michael Wonder on 06 Jun 2019
Enzyvant announces FDA acceptance of biologics license application and priority review status for RVT-802, a novel investigational tissue-based regenerative therapy for paediatric congenital athymia
5 June 2019 - RVT-802, a one-time therapy, leverages Enzyvant’s T cell generation platform designed to treat profound immunodeficiencies. ...
Posted by Michael Wonder on 04 Jun 2019
US FDA review for Scenesse extended by three months
3 June 2019 - The FDA sets new goal date of 6 October 2019 to complete review and issue risk-benefit decision. ...
Posted by Michael Wonder on 08 May 2019
FDA undercuts $375,000 drug in surprise move
8 May 2019 - The US FDA created a workaround this week that effectively undercuts the $375,000 price tag of ...
Posted by Michael Wonder on 08 May 2019
Akcea and Ionis announce approval of Waylivra (volanesorsen) in the European Union
7 May 2019 - Akcea Therapeutics and Ionis Pharmaceuticals announced today that Waylivra has received conditional marketing authorisation from the ...