Blog
RSS FeedPosted by Michael Wonder on 03 Aug 2024
FDA has accepted a BLA for bentracimab, the first and only ticagrelor reversal agent, for filing and priority review
2 August 2024 - SFJ Pharmaceuticals, sponsor of the bentracimab biologics license application, and SERB Pharmaceuticals (SERB), who acquired exclusive ...
Posted by Michael Wonder on 02 Aug 2024
Adverum Biotechnologies announces FDA regenerative medicine advanced therapy designation granted for ixo-vec for the treatment of wet AMD
1 August 2024 - Adverum Biotechnologies today announced that the US FDA has granted regenerative medicine advanced therapy designation for ...
Posted by Michael Wonder on 30 Jul 2024
AffyImmune receives FDA regenerative medicine advanced therapy designation for AIC100 in recurrent anaplastic thyroid cancer
23 July 2024 - RMAT designation follows FDA’s review of available preliminary clinical evidence that AIC100 has the potential to address ...
Posted by Michael Wonder on 30 Jul 2024
Novartis Scemblix granted FDA priority review for the treatment of adults with newly diagnosed CML
29 July 2024 - Priority review based on ASC4FIRST Phase 3 study with Scemblix data first to show significantly improved molecular ...
Posted by Michael Wonder on 25 Jul 2024
ConSynance Therapeutics receives FDA rare paediatric disease designation for CSTI-500 in Prader-Willi syndrome
24 July 2024 - ConSynance Therapeutics today announced that the US FDA has granted rare paediatric disease designation to its ...
Posted by Michael Wonder on 20 Jul 2024
Neuren receives rare paediatric disease designation from FDA
19 July 2024 - Neuren Pharmaceuticals has received rare paediatric disease designation from the US FDA for NNZ-2591 in Phelan-McDermid ...
Posted by Michael Wonder on 19 Jul 2024
Atara Biotherapeutics announces US FDA acceptance and priority review of the biologics license application for tabelecleucel for the treatment of Epstein-Barr virus positive post-transplant lymphoproliferative disease
17 July 2024 - PDUFA target action date of 15 January 2025. ...
Posted by Michael Wonder on 18 Jul 2024
Sellas announces US FDA rare paediatric disease designation granted to SLS009 for the treatment of paediatric acute myeloid leukaemia
16 July 2024 - Opened enrollment for paediatric AML patients in on-going Phase 2 clinical trial. ...
Posted by Michael Wonder on 13 Jul 2024
Innorna announces US FDA rare paediatric disease designation granted to IN016 for the treatment of progressive familial intrahepatic cholestasis
11 July 2024 - Innorna today announced that the US FDA has granted rare paediatric disease designation to IN016, one of ...
Posted by Michael Wonder on 03 Jul 2024
The rare paediatric disease voucher program creates new treatments. I have new data to prove it.
2 July 2024 - The FDA’s rare paediatric disease priority review voucher program, which has been providing incentives for lifesaving innovations ...
Posted by Michael Wonder on 01 Jul 2024
Neurocrine Biosciences announces US FDA accepts new drug applications and grants priority review for crinecerfont for paediatric and adult patients with CAH
1 July 2024 - PDUFA target action dates in late December 2024. ...
Posted by Michael Wonder on 27 Jun 2024
Ionis announces olezarsen FCS new drug application accepted for priority review
26 June 2024 - Olezarsen PDUFA date set for 19 December 2024 for treatment of familial chylomicronemia syndrome. ...
Posted by Michael Wonder on 25 Jun 2024
Sellas announces US FDA rare paediatric disease designation granted to SLS009 for the treatment of paediatric acute lymphoblastic leukaemia
24 June 2024 - Sellas Life Sciences today announced that the US FDA has granted rare paediatric disease designation to SLS009, ...
Posted by Michael Wonder on 23 Jun 2024
Neurotech Pharmaceuticals receives priority review of biologics license application for NT-501 (revakinagene taroretcel) as a treatment for macular telangiectasia type 2
20 June 2024 - Priority review granted with a PDUFA goal date set for 17 December 2024. ...
Posted by Michael Wonder on 20 Jun 2024
Aurion Biotech receives breakthrough therapy designation and regenerative medicine advanced therapy designation for its drug candidate AURN001
19 June 2024 - First allogeneic cell therapy to receive both FDA designations for the treatment of corneal oedema secondary to ...