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RSS FeedPosted 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 ...
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 ...
Posted by Michael Wonder on 10 Jun 2024
Tagrisso granted priority review in the US for patients with unresectable, stage III EGFR mutated lung cancer
10 June 2024 - Decision based on LAURA Phase 3 trial results which extended median progression-free survival by more than ...
Posted by Michael Wonder on 06 Jun 2024
BlueRock Therapeutics receives FDA Regenerative Medicine Advanced Therapy designation for Parkinson’s disease cell therapy candidate bemdaneprocel
31 May 2024 - Regenerative Medicine Advanced Therapy designation follows phase I clinical trial results demonstrating that bemdaneprocel is well tolerated ...
Posted by Michael Wonder on 06 Jun 2024
Neurogene announces NGN-401 gene therapy for Rett syndrome selected by FDA for START pilot program
3 June 2024 - NGN-401 is one of only three CBER programs chosen by FDA. ...
Posted by Michael Wonder on 06 Jun 2024
Denali Therapeutics announces FDA has selected DNL126 for MPS IIIA (Sanfilippo syndrome type A) for START pilot program intended to accelerate development of rare disease therapies
3 June 2024 - START is a pilot program newly launched jointly by the FDA CDER and CBER divisions to further ...
Posted by Michael Wonder on 06 Jun 2024
Moderna's investigational therapeutic for methylmalonic acidaemia (mRNA-3705) selected by US FDA for START pilot program
6 June 2024 - Center for Biologics Evaluation and Research has chosen mRNA-3705 as one of four investigational medicines for ...
Posted by Michael Wonder on 05 Jun 2024
TScan Therapeutics receives FDA’s regenerative medicine advanced therapy designation for its two lead TCR-T therapy candidates for the treatment of heme malignancies
29 May 2024 - RMAT designation granted for both TSC-100 and TSC-101 for the treatment of patients with AML, ALL, and ...