PBAC Public Summary Documents (first time decisions not to recommend, deferrals and other matters) – May 2021 meeting

10 September 2021 - The Public Summary Documents (first time decisions not to recommend, deferrals and other matters) from the May ...

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Decibel Therapeutics receives orphan drug and rare paediatric disease designations for DB-OTO for the treatment of otoferlin related congenital hearing loss

9 September 2021 - Decibel Therapeutics today announced that the U.S. FDA has granted both orphan drug designation and rare paediatric ...

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UK MHRA grants GenSight Biologics’ Lumevoq ophthalmic gene therapy Promising Innovative Medicine Designation

6 September 2021 - GenSight Biologics today announced that its gene therapy Lumevoq has been granted Promising Innovative Medicine designation by ...

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Implementing outcomes based managed entry agreements for rare disease treatments: nusinersen and tisagenlecleucel

10 August 2021 - Enthusiasm for the use of outcomes based managed entry agreements to manage uncertainties apparent at the time ...

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Mustang Bio receives European Medicines Agency PRIME designation for MB-107 to treat X-linked severe combined immunodeficiency in newly diagnosed infants

2 August 2021 - Mustang Bio today announced that the EMA has granted Priority Medicines designation to MB-107, its lentiviral ...

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Family says choice is life in Australia or death waiting for PHARMAC

2 August 2021 - A Tauranga couple is splitting up their family so their daughter can access treatment for spinal muscular ...

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EMA validates BioMarin's marketing authorisation application for valoctocogene roxaparvovec to treat severe haemophilia A

15 July 2021 - Potential first gene therapy in Europe for treatment of haemophilia A. ...

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Novartis Korea to continue discussing reimbursement plan for SMA drug Zolgensma

9 July 2021 - Novartis said Friday that it would continue to discuss with the domestic regulators to receive reimbursement ...

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Lysogene announces FDA fast track designation for LYS-GM101 gene therapy for the treatment of GM1 gangliosidosis

8 July 2021 - Lysogene today announced that the U.S. FDA has granted fast track designation to its LYS-GM101 program, which ...

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BioMarin resubmits marketing authorisation application to European Medicines Agency for valoctocogene roxaparvovec to treat severe haemophilia A

28 June 2021 - Valoctocogene roxaparvovec MAA granted request for accelerated assessment. ...

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AskBio receives FDA fast track designation for LION-101, a novel investigational AAV gene therapy for the treatment of limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9)

28 June 2021 - LGMD2I/R9 is a rare form of muscular dystrophy with no approved therapies. ...

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Targovax receives fast track designation for ONCOS-102 in melanoma

22 June 2021 - Targovax today announces that its lead clinical candidate ONCOS-102 has received fast track designation in PD-1-refractory ...

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Ireland an outlier without access to new cell and gene therapy, report says

27 May 2021 - Upfront costs high but treatments may cut long-term expense and boost patient results. ...

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Colchester baby's parents feel 'abandoned' over £1.7 million drug

25 May 2021 - A baby with a fatal genetic condition has been "abandoned" in a "race against time" for ...

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European Medicines Agency grants BioMarin's request for accelerated assessment of valoctocogene roxaparvovec for treatment of severe haemophilia A

24 May 2021 - MAA resubmission on track for June 2021. ...

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