Blog
RSS FeedPosted by Michael Wonder on 05 Aug 2019
Alnylam announces U.S. FDA granted priority review of the givosiran new drug application for the treatment of acute hepatic porphyria
5 August 2019 - PDUFA date set for 4 February 2020. ...
Posted by Michael Wonder on 31 Jul 2019
Supporting medicine developers in generating quality data packages in early access approaches (PRIME and breakthrough therapies)
31 July 2019 - EMA and the US FDA have published today a report on their joint workshop with stakeholders ...
Posted by Michael Wonder on 29 Jul 2019
FDA updates draft guidance on rare paediatric disease priority review voucher program
29 July 2019 - The U.S. FDA today issued a revised draft guidance, Rare Paediatric Disease Priority Review Vouchers – ...
Posted by Michael Wonder on 26 Jul 2019
Matinas BioPharma receives qualified infectious disease product and fast track designations from U.S. FDA for MAT2203 for the treatment of cryptococcal meningitis
25 July 2019 - Fourth QIDP and fast track designations granted by FDA for MAT2203. ...
Posted by Michael Wonder on 18 Jul 2019
LifeMax receives rare paediatric disease designation for LM-030 for the treatment of Netherton syndrome
18 July 2019 - LifeMax Laboratories today announced that the FDA granted rare paediatric disease designation to LM-030, an investigational therapy ...
Posted by Michael Wonder on 17 Jul 2019
LogicBio Therapeutics receives rare paediatric disease designation for LB-001 for the treatment of methylmalonic acidaemia
16 July 2019 - LogicBio Therapeutics today announced the U.S. FDA has granted rare paediatric disease designation to LB-001, a ...
Posted by Michael Wonder on 16 Jul 2019
FDA accepts file and accelerates review of Novartis sickle cell disease medicine crizanlizumab
16 July 2019 - FDA grants crizanlizumab Priority Review based on Phase II data showing prevention of vaso-occlusive crises in patients ...
Posted by Michael Wonder on 09 Jul 2019
ViiV Healthcare announces start of first-ever study to identify and evaluate approaches to implementing its once-monthly injectable HIV treatment in clinical practice
9 July 2019 - The long-acting injectable regimen has been granted priority review status by the FDA, with a target approval ...
Posted by Michael Wonder on 25 Jun 2019
U.S. FDA accepts GSK’s application for Zejula (niraparib) in late stage ovarian cancer with priority review
24 June 2019 - GlaxoSmithKline plc today announced that Tesaro, an oncology-focused business acquired by GSK, submitted a supplemental new ...
Posted by Michael Wonder on 25 Jun 2019
Krystal Biotech receives regenerative medicine advanced therapy designation from FDA for KB103
24 June 2019 - RMAT designation granted to KB103 based on positive interim data from GEM-1 and GEM-2 study. ...
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 13 Jun 2019
FDA grants priority review to Genentech’s Rituxan (rituximab) in children with two rare blood vessel disorders
12 June 2019 - If approved, this would be the first paediatric indication for Rituxan. ...
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 03 Jun 2019
Galt Pharmaceuticals answers the call for better practices in pain management with a non-opioid therapeutic alternative
29 May 2019 - Officials with the FDA have given priority review designation to an opioid-free pain management treatment from ...
Posted by Michael Wonder on 31 May 2019
U.S. FDA grants priority review for daratumumab in combination with bortezomib, thalidomide and dexamethasone in frontline multiple myeloma
30 May 2019 - 26 September 2019 PDUFA date. ...