Top FDA official: agency needs to start using accelerated approval for gene therapies

20 March 2023 - A top FDA official said Monday that the agency needs to start using accelerated approval, a ...

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BioMarin provides update on FDA review of Roctavian (valoctocogene roxaparvovec) gene therapy for adults with severe haemophilia A

7 March 2023 - FDA extends PDUFA target action date to 30 June 2023. ...

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Alnylam announces US FDA acceptance of supplemental new drug application for Onpattro (patisiran) for the treatment of the cardiomyopathy of ATTR amyloidosis

21 February 2023 - PDUFA date set for 8 October 2023. ...

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FDA gene therapy head raises concern on accelerated approval use

14 February 2023 - More than 1,000 cell and gene therapy clinical trials registered. ...

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Top FDA official interested in ‘Project Orbis’ for cell and gene therapies

13 February 2023 - A top official from the US FDA said he wants to see something similar to the agency’s ...

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Rocket Pharmaceuticals receives FDA regenerative medicine advanced therapy designation for RP-A501 gene therapy for Danon disease

7 February 2023 - Rocket Pharmaceuticals today announced that the US FDA has granted regenerative medicine advanced therapy designation to ...

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Nanoscope Therapeutics receives fast track designation by the FDA for MCO-010 for the treatment of Stargardt disease

30 January 2023 - Nanoscope Therapeutics today announced that the US FDA has granted fast track designation to MCO-010, an ...

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FDA approves first gene therapy for the treatment of high risk, non-muscle invasive bladder cancer

16 December 2022 - Today, the US FDA approved Adstiladrin (nadofaragene firadenovec-vncg), a non-replicating adenoviral vector based gene therapy indicated for ...

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How CSL priced the world’s most expensive drug

1 December 2022 - When blood products giant CSL announced last week it had been given US regulatory approval for ...

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Sarepta Therapeutics announces that US FDA has accepted for filing and granted priority review for the biologics license application for SRP-9001, Sarepta’s gene therapy for the treatment of ambulant individuals with Duchenne muscular dystrophy

28 November 2022 - Regulatory action date of 29 May 2023. ...

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BioMarin announces advancements in FDA review of Roctavian (valoctocogene roxaparvovec) for adults with severe haemophilia A

23 November 2022 - FDA no longer plans to hold an advisory committee meeting, as previously planned, to discuss the biologics ...

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CSL lands world first gene therapy with $5.3 million haemophilia treatment

23 November 2022 - Australian biotech giant CSL will bring the first gene therapy for haemophilia B to market in ...

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FDA approves first gene therapy to treat adults with haemophilia B

22 November 2022 - Today, the US FDA approved Hemgenix (etranacogene dezaparvovec), an adeno-associated virus vector-based gene therapy for the treatment ...

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BioMarin announces incremental progress on biologics license application review for valoctocogene roxaparvovec AAV gene therapy for adults with severe haemophilia A program

7 November 2022 - FDA requests submission of upcoming 3 year data analysis from Phase 3 GENEr8-1 trial. ...

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Sensorion announces US FDA grants rare paediatric disease designation to OTOF-GT for the treatment of otoferlin gene-mediated hearing loss

7 November 2022 - OTOF-GT targets the restoration of hearing in people living with otoferlin deficiency. ...

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