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RSS FeedPosted 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 01 Jul 2024
Sipavibart EMA regulatory submission accepted under accelerated assessment for COVID-19 prevention
1 July 2024 - Submission based on positive SUPERNOVA Phase 3 trial data which demonstrated a statistically significant reduction in ...
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 26 Jun 2024
ExCellThera announces EMA’s acceptance under accelerated assessment of market authorisation application for UM171 cell therapy for patients with haematological malignancies who lack a readily available suitable donor
25 June 2024 - ExCellThera announced today that the market authorisation application for UM171 cell therapy (INN-dorocubicel) has been accepted under ...
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 ...
Posted by Michael Wonder on 18 Jun 2024
US FDA rare paediatric disease designation granted to RC220 bisantrene for the treatment of paediatric AML
18 June 2024 - Race Oncology is pleased to announce that the US FDA has extended rare paediatric disease designation ...
Posted by Michael Wonder on 11 Jun 2024
Calico Life Sciences announces that fosigotifator (ABBV-CLS-7262) for vanishing white matter disease has been selected for the FDA START pilot program
7 June 2024 -- Calico Life Sciences today announced that its investigational eIF2B activator fosigotifator (ABBV-CLS-7262) has been accepted into ...