Blog
RSS FeedPosted by Michael Wonder on 24 Sep 2020
bluebird bio’s LentiGlobin for sickle cell disease gene therapy (bb1111) granted priority medicines (PRIME) designation by European Medicines Agency
23 September 2020 - EMA’s PRIME program designed to optimise development and expedite evaluation of innovative medicines for patients with high ...
Posted by Michael Wonder on 16 Sep 2020
GenSight Biologics submits EU marketing authorisation application for Lumevoq gene therapy to treat vision loss due to Leber hereditary optic neuropathy
15 September 2020 - First marketing authorisation application for a gene therapy treating a mitochondrial disease. ...
Posted by Michael Wonder on 10 Sep 2020
EU review extended for BioMarin's gene therapy Valrox
10 September 2020 - European regulators have requested more data to review the marketing application for BioMarin's haemophilia A gene ...
Posted by Michael Wonder on 19 May 2020
AveXis receives EC approval and activates “Day One” access program for Zolgensma, the only gene therapy for spinal muscular atrophy
19 May 2020 - Zolgensma (onasemnogene abeparvovec) is conditionally approved in Europe for the treatment of patients with spinal muscular atrophy ...
Posted by Michael Wonder on 28 Mar 2020
AveXis receives positive CHMP opinion for Zolgensma, the only gene therapy for spinal muscular atrophy
28 March 2020 - AveXis, a Novartis company, today announced that the CHMP of the EMA has adopted a positive ...
Posted by Michael Wonder on 04 Mar 2020
Alnylam announces approval of Givlaari (givosiran) in the European Union for the treatment of acute hepatic porphyria in adults and adolescents
3 March 2020 - Givlaari approved based on ENVISION Phase 3 study results showing a 74% reduction in the rate of ...
Posted by Michael Wonder on 03 Mar 2020
MeiraGTx announces Priority Medicines (PRIME) and Advanced Therapy Medicinal Product designations granted by the EMA to AAV-RPGR gene therapy for the treatment of X-linked retinitis pigmentosa
2 March 2020 - PRIME designation granted based on clinical data from ongoing Phase 1/2 trial of AAV-RPGR. ...
Posted by Michael Wonder on 03 Feb 2020
Alnylam receives positive CHMP opinion for Givlaari (givosiran) for the treatment of acute hepatic porphyria in adults and adolescents
31 January 2020 - Positive opinion is based on data from the pivotal ENVISION Phase 3 study. ...
Posted by Michael Wonder on 01 Feb 2020
Highlights from the CHMP 27-30 January 2020 meeting
31 January 2020 - Fifteen new medicines recommended for approval. ...
Posted by Michael Wonder on 14 Jan 2020
PTC Therapeutics provides corporate update
13 January 2020 - PTC-AADC MAA submitted; BLA now expected to be submitted in 2Q 2020. ...
Posted by Michael Wonder on 24 Dec 2019
European Medicines Agency validates BioMarin's marketing authorisation application for valoctocogene roxaparvovec to treat severe haemophilia A
23 December 2019 - Potential first gene therapy in Europe directed at any type of haemophilia. ...
Posted by Michael Wonder on 17 Dec 2019
Rocket Pharmaceuticals receives EMA PRIME eligibility for RP-L102 gene therapy for Fanconi anaemia
16 December 2019 - Milestone gives Fanconi anaemia program all accelerated regulatory tools in U.S. and EU, including FDA regenerative medicine ...
Posted by Michael Wonder on 22 Nov 2019
BioMarin submits marketing authorisation application to EMA for valoctocogene roxaparvovec to treat severe haemophilia A
21 November 2019 - First marketing application submission for gene therapy directed at any type of haemophilia. ...
Posted by Michael Wonder on 13 Nov 2019
Regulators’ advice can make a difference for faster patient access to highly innovative therapies
12 November 2019 - Early dialogue and frequent interactions between medicine developers, regulators, health technology assessment bodies and patients can play ...
Posted by Michael Wonder on 06 Nov 2019
One shot drug to end Sicilian curse comes at $1.8 million cost
5 November 2019 - A new targeted gene therapy shows the promise and pitfalls of medical advances. ...