Blog
RSS FeedPosted 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. ...
Posted by Michael Wonder on 30 Jul 2019
Novartis’ Zolgensma joins growing list of medicines to lose accelerated assessment status in EU
29 July 2019 - The EMA's CHMP recently announced its decision to remove Novartis’ spinal muscular atrophy gene therapy onasemnogene ...
Posted by Michael Wonder on 30 Jul 2019
EMA grants PRIME access to ProQR’s sepofarsen for Leber’s congenital amaurosis 10
29 July 2019 - Access based on positive interim analysis of clinical data as well as preclinical data to date. ...
Posted by Michael Wonder on 02 Jul 2019
Alnylam submits marketing authorisation application to the EMA for givosiran for the treatment of acute hepatic porphyria
1 July 2019 - Patients receiving givosiran in pivotal Phase 3 ENVISION study had a 74% mean reduction in annualised rate ...
Posted by Michael Wonder on 15 Jun 2019
Bluebird Bio CEO defends $1.8 million price for gene therapy — ‘It’s really thinking about it differently’
14 June 2019 - He says the one-time treatment is a game changer for patients, giving them a “lifelong benefit” while ...
Posted by Michael Wonder on 04 Jun 2019
bluebird bio announces EU conditional marketing authorisation for Zynteglo (autologous CD34+ cells encoding βA-T87Q-globin gene) gene therapy for patients 12 years and older with transfusion-dependent β-thalassemia who do not have β0/β0 genotype
3 June 2019 - European marketing authorisation for Zynteglo follows the fastest assessment of an advanced therapy medicinal product as part ...
Posted by Michael Wonder on 08 May 2019
Akcea and Ionis announce approval of Waylivra (volanesorsen) in the European Union
7 May 2019 - Akcea Therapeutics and Ionis Pharmaceuticals announced today that Waylivra has received conditional marketing authorisation from the ...
Posted by Michael Wonder on 01 Apr 2019
OncoSec announces advanced therapy medicinal product classification from the EMA for TAVO in refractory metastatic melanoma
1 April 2019 - Classification as an ATMP Positions TAVO for accelerated review and approval in Europe. ...
Posted by Michael Wonder on 04 Mar 2019
Akcea and Ionis receive positive EU CHMP opinion for Waylivra (volanesorsen)
1 March 2019 - First and only therapy approved for FCS, a serious and rare disease with no approved treatment options. ...
Posted by Michael Wonder on 23 Nov 2018
Novartis's pricing might be tested with costly eye therapy
23 November 2018 - Novartis’s price-setting muscle is about to be tested after it won European Union approval for a blindness-fighting ...
Posted by Michael Wonder on 23 Nov 2018
Novartis announces landmark EU approval for one-time gene therapy Luxturna to restore vision in people with rare inherited retinal disease
23 November 2018 - Luxturna (voretigene neparvovec) is the first gene therapy to treat an inherited retinal disease, indicated for children ...
Posted by Michael Wonder on 06 Oct 2018
bluebird bio announces EMA’s acceptance of marketing authorisation application for lentiGlobin gene therapy for the treatment of transfusion-dependent β-thalassemia
5 October 2018 - EMA will evaluate lentiGlobin marketing authorisation application under accelerated assessment. ...
Posted by Michael Wonder on 05 Oct 2018
Orchard Therapeutics receives EMA PRIME designation for OTL-300, an investigational lentiviral gene therapy for the treatment of transfusion-dependent beta thalassaemia
4 October 2018 - Orchard Therapeutics today announced that the EMA has granted Priority Medicines (PRIME) designation to OTL-300, an ...