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RSS FeedPosted by Michael Wonder on 10 Sep 2021
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 ...
Posted by Michael Wonder on 10 Sep 2021
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 ...
Posted by Michael Wonder on 06 Sep 2021
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 ...
Posted by Michael Wonder on 10 Aug 2021
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 ...
Posted by Michael Wonder on 03 Aug 2021
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 ...
Posted by Michael Wonder on 01 Aug 2021
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 ...
Posted by Michael Wonder on 16 Jul 2021
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. ...
Posted by Michael Wonder on 11 Jul 2021
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 ...
Posted by Michael Wonder on 08 Jul 2021
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 ...
Posted by Michael Wonder on 29 Jun 2021
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. ...
Posted by Michael Wonder on 28 Jun 2021
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. ...
Posted by Michael Wonder on 22 Jun 2021
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 ...
Posted by Michael Wonder on 27 May 2021
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. ...
Posted by Michael Wonder on 26 May 2021
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 ...
Posted by Michael Wonder on 25 May 2021
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. ...