Blog
RSS FeedPosted by Michael Wonder on 06 Dec 2022
Ionis announces European Medicines Agency accepts marketing authorisation application of tofersen to treat rare, genetic form of ALS
5 December 2022 - EMA acceptance follows FDA's acceptance of tofersen new drug application earlier this year. ...
Posted by Michael Wonder on 21 Sep 2022
Alnylam receives approval in Europe for Amvuttra (vutrisiran) for the treatment of hereditary transthyretin-mediated amyloidosis in adult patients with stage 1 or stage 2 polyneuropathy
20 September 2022 - Amvuttra demonstrated halting or reversal in neuropathy impairment with subcutaneous administration once every three months. ...
Posted by Michael Wonder on 25 Aug 2022
First gene therapy for adults with severe haemophilia A, BioMarin's Roctavian (valoctocogene roxaparvovec), approved by European Commission
24 August 2022 - Maintains orphan drug designation in the EU providing 10 years of market exclusivity. ...
Posted by Michael Wonder on 23 Jul 2022
Alnylam receives positive CHMP opinion for vutrisiran for the treatment of hereditary transthyretin-mediated amyloidosis in adult patients with stage 1 or stage 2 polyneuropathy
22 July 2022 - Positive opinion based on HELIOS-A Phase 3 study. ...
Posted by Michael Wonder on 28 Jun 2022
Adverum Biotechnologies granted Priority Medicines (PRIME) designation by EMA for ADVM-022 in wet AMD
24 June 2022 - Adverum Biotechnologies today announced that the EMA has granted ADVM-022 Priority Medicines (PRIME) designation for the treatment ...
Posted by Michael Wonder on 26 Jun 2022
First gene therapy to treat severe haemophilia A
24 June 2022 - EMA has recommended granting a conditional marketing authorisation in the European Union (EU) for Roctavian (valoctocogene roxaparvovec) ...
Posted 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 ...