Blog
RSS FeedPosted by Michael Wonder on 25 Jan 2022
Genentech’s Evrysdi (risdiplam) granted FDA priority review for treatment of presymptomatic babies under 2 months of age with spinal muscular atrophy
24 January 2022 - Interim data submitted to the FDA show majority of pre-symptomatic babies treated with Evrysdi for at least ...
Posted by Michael Wonder on 18 Jan 2022
bluebird provides update on FDA review timelines for betibeglogene autotemcel for beta thalasszemia and elivaldogene autotemcel for cerebral adrenoleukodystrophy
18 January 2022 - FDA PDUFA goal dates for both therapies extended by three months. ...
Posted by Michael Wonder on 11 Jan 2022
4D Molecular Therapeutics announces FDA fast track designation granted to 4D-125 for the treatment of X-linked retinitis pigmentosa
10 January 2022 - 4D Molecular Therapeutics announced that the U.S. FDA has granted fast track designation for 4D-125 for treatment ...
Posted by Michael Wonder on 26 Dec 2021
Public Summary Documents – September 2021 PBAC meeting
24 December 2021 - The Public Summary Documents (positive recommendations and subsequent decisions not to recommend) from the September 2021 PBAC ...
Posted by Michael Wonder on 18 Dec 2021
bluebird bio announces FDA priority review of biologics license application for eli-cel gene therapy for cerebral adrenoleukodystrophy in patients without a matched sibling donor
17 December 2021 - If approved, eli-cel will be the first and only gene therapy for the treatment of cerebral adrenoleukodystrophy, ...
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 14 Dec 2021
EU regulation passed: G-BA works on European HTA assessment
14 December 2021 - Today the EU Parliament passed the EU-HTA regulation for a joint health technology assessment, which includes, ...
Posted by Michael Wonder on 24 Nov 2021
ICER to assess gene therapy for beta thalassaemia
24 November 2021 - Report will be subject of New England CEPAC meeting in June 2022; draft scoping document open ...
Posted by Michael Wonder on 22 Nov 2021
bluebird bio Announces FDA priority review of biologics license application for beti-cel gene therapy for patients with β-thalassemia who require regular red blood cell transfusions
22 November 2021 - FDA set PDUFA date of 20 May 2022. ...
Posted by Michael Wonder on 08 Nov 2021
Drugs against rare diseases: Libmeldy gene therapy shows additional benefits - Zolgensma cell therapy, on the other hand, does not
4 November 2021 - The Federal Joint Committee (G-BA) today assessed the additional benefit of two active ingredients against very rare, ...
Posted by Michael Wonder on 03 Nov 2021
AvroBio receives rare paediatric disease designation from the U.S. FDA for AVR-RD-05, a gene therapy for mucopolysaccharidosis type II or Hunter syndrome
3 November 2021 - AVROBIO today announced that the U.S. FDA has granted rare paediatric disease designation to AVR-RD-05, its ...
Posted by Michael Wonder on 27 Oct 2021
FDA, NIH and 15 private organisations join forces to increase effective gene therapies for rare diseases
27 October 2021 - The U.S. FDA, the National Institutes of Health, 10 pharmaceutical companies and five non-profit organisations have partnered ...
Posted by Michael Wonder on 23 Oct 2021
Sio Gene Therapies announces granting of FDA fast track designation for investigational AXO-AAV-GM1 (AAV9-GLB1) gene therapy in patients with GM1 gangliosidosis
21 October 2021 - Sio Gene Therapies today announced that the U.S. FDA has granted fast track designation to AXO-AAV-GM1, its ...
Posted by Michael Wonder on 21 Oct 2021
NICE recommends gene silencing therapy for porphyria patients on NHS
21 October 2021 - NICE have recommended the use of givosiran, a gene silencing therapy, on the NHS in England, as ...
Posted by Michael Wonder on 18 Oct 2021
Novartis applauds Quebec, the first province to list Zolgensma for the treatment of paediatric patients with spinal muscular atrophy
15 October 2021 - Public reimbursement under the Régie de l'assurance maladie du Québec, effective October 20th will apply for children ...