Orchard Therapeutics announces Swissmedic validation of the marketing authorisation application for Libmeldy (atidarsagene autotemcel)

1 December 2022 - Orchard Therapeutics today announced its marketing authorisation application for Libmeldy (atidarsagene autotemcel) has been accepted 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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ICER to assess gene therapies for sickle cell disease

23 November 2022 - Report will be subject of CTAF meeting in June 2023; draft scoping document open to public ...

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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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FDA finalises umbrella trial guidance for cell and gene therapies

4 November 2022 - The US FDA laid out its recommendations for sponsors to study multiple versions of a cellular or ...

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ICER publishes evidence report on gene therapies for haemophilia A and B

2 November 2022 - Duration of benefit with gene therapies and risks for rare side effects remain major uncertainties. ...

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AvroBio receives rare paediatric disease designation from the US FDA for first in class gene therapy for Gaucher disease

27 October 2022 - AVR-RD-02 has previously received fast track status from FDA, orphan drug designation in the US and EU, ...

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AvroBio granted ILAP designation from UK MHRA for first in class gene therapy for Gaucher disease

18 October 2022 - ILAP intended to accelerate regulatory review process and facilitate patient access in UK for seriously debilitating and ...

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Biogen announces FDA’s 3 month extension of review period for the new drug application for tofersen

17 October 2022 - The new Prescription Drug User Fee Act action date set by the FDA is 25 April ...

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Gene therapy Luxturna now reimbursed in Quebec for people with previously untreatable inherited vision loss

17 October 2022 - Quebec leads the way as the first province to reimburse Luxturna (voretigene neparvovec) for previously untreatable inherited ...

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