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 ...

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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. ...

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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. ...

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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 ...

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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 ...

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Vertex announces FDA acceptance of new drug application for vanzacaftor/tezacaftor/deutivacaftor, a next in class triple combination treatment for cystic fibrosis

2 July 2024 - Vanza triple granted priority review with a PDUFA target action date of 2 January 2025. ...

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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. ...

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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 ...

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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. ...

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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 ...

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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, ...

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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. ...

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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 ...

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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 ...

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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 ...

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