Blog
RSS FeedPosted by Michael Wonder on 21 May 2022
PTC Therapeutics receives positive CHMP opinion for Upstaza for the treatment of AADC deficiency
20 May 2022 - First ever gene therapy directly administered into the brain. ...
Posted by Michael Wonder on 29 Mar 2022
European Medicines Agency commences review of novel gene therapy candidate etranacogene dezaparvovec for people with haemophilia B
29 March 2022 - Marketing authorisation application for etranacogene dezaparvovec will be reviewed under accelerated assessment and has the potential to ...
Posted by Michael Wonder on 25 Mar 2022
New gene therapy to treat adult patients with multiple myeloma
25 March 2022 - EMA has recommended a conditional marketing authorisation in the European Union for Carvykti (ciltacabtagene autoleucel) for the ...
Posted by Michael Wonder on 16 Dec 2021
CSL Behring receives accelerated CHMP assessment for etranacogene dezaparvovec for European patients living with haemophilia B
15 December 2021 - CSL Behring today announced that the CHMP, the chief scientific body of the EMA accepted its ...
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 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 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 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. ...
Posted by Michael Wonder on 23 May 2021
First gene therapy to treat children with rare inherited neurological disease
21 May 2021 - The EMA has recommended granting a marketing authorisation in the European Union for the gene therapy ...
Posted by Michael Wonder on 19 Nov 2020
Alnylam receives approval for Oxlumo (lumasiran) in the European Union for the treatment of primary hyperoxaluria type 1 in all age groups
19 November 2020 - Oxlumo is the first therapeutic approved for the treatment of primary hyperoxaluria type 1, and the ...
Posted by Michael Wonder on 03 Nov 2020
GenSight Biologics reports validation of Lumevoq marketing authorisation application by European Medicines Agency
3 November 2020 - GenSight Biologics today reported that the Lumevoq marketing authorisation application passed the validation checks required for submissions ...
Posted by Michael Wonder on 18 Oct 2020
Highlights from 12-15 October CHMP meeting
16 October 2020 - Ten new medicines recommended for approval. ...
Posted by Michael Wonder on 18 Oct 2020
Novartis receives positive CHMP opinion for Leqvio (inclisiran), a potential first-in-class siRNA for the treatment of high cholesterol
16 October 2020 - If approved, inclisiran will be the first and only small interfering RNA (siRNA) in Europe for patients ...
Posted by Michael Wonder on 18 Oct 2020
First treatment for rare condition primary hyperoxaluria type 1
16 October 2020 - EMA has recommended granting a marketing authorisation in the European Union for Oxlumo (lumasiran) for the ...
Posted by Michael Wonder on 04 Oct 2020
bluebird bio announces European Medicines Agency’s acceptance of marketing authorisation application for elivaldogene autotemcel (eli-cel, Lenti-D) gene therapy for cerebral adrenoleukodystrophy
2 October 2020 - European Medicines Agency will evaluate eli-cel application under accelerated assessment. ...