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RSS FeedPosted by Michael Wonder on 11 Jul 2023
Synlogic granted fast track designation from FDA for labafenogene marselecobac (SYNB1934) for treatment of phenylketonuria
11 July 2023 - Synlogic today announced that the US FDA has granted fast track designation to labafenogene marselecobac (previously known ...
Posted by Michael Wonder on 07 Jul 2023
FDA grants rare paediatric disease designation to NS-089/NCNP-02 for the treatment of Duchenne muscular dystrophy
7 July 2023 - NS Pharma announced today the US FDA has granted rare paediatric disease designation to NS-089/NCNP-02 (brogidirsen) an ...
Posted by Michael Wonder on 03 Jul 2023
BioMarin’s next Roctavian challenge is how to successfully commercialise its gene therapy: warranties may be an option
1 July 2023 - After quite the regulatory process, with Roctavian (valoctocogene roxaparvovec) BioMarin has a potential blockbuster. On 29 ...
Posted by Michael Wonder on 30 Jun 2023
FDA approves ARUP Laboratories' AAV5 DetectCDx, a first-ever companion diagnostic immunoassay for a gene therapy
29 June 2023 - FDA issues simultaneous approval of Roctavian (valoctocogene roxaparvovec-rvox), BioMarin's gene therapy for severe haemophilia A. ...
Posted by Michael Wonder on 29 Jun 2023
US FDA approves BioMarin's Roctavian (valoctocogene roxaparvovec-rvox), the first and only gene therapy for adults with severe haemophilia A
29 June 2023 - Roctavian's approval was based on durability, efficacy and safety results from the largest and longest Phase 3 ...
Posted by Michael Wonder on 27 Jun 2023
FDA accepts Pfizer’s application for haemophilia B gene therapy fidanacogene elaparvovec
27 June 2023 - Submissions based on positive Phase 3 data from BENEGENE-2 trial. ...
Posted by Michael Wonder on 22 Jun 2023
FDA approves first gene therapy for treatment of certain patients with Duchenne muscular dystrophy
22 June 2023 - Today, the US FDA approved Elevidys, the first gene therapy for the treatment of pediatric patients 4 ...
Posted by Michael Wonder on 21 Jun 2023
bluebird bio announces FDA priority review of the biologics license application for lovotibeglogene autotemcel (lovo-cel) for patients with sickle cell disease 12 years and older with a history of vaso-occlusive events
21 June 2023 - PDUFA date set for 20 December 2023. ...
Posted by Michael Wonder on 20 Jun 2023
CSL Behring announces the first patient has received FDA approved Hemgenix (etranacogene dezaparvovec-drlb) for haemophilia B
20 June 2023 - Hemgenix, the first and only FDA approved gene therapy for haemophilia B, has been proven to ...
Posted by Michael Wonder on 15 Jun 2023
FDA should reject accelerated approval of gene therapy SRP-9001
15 June 2023 - The US FDA should reject the accelerated approval of the gene therapy SRP-9001 (Sarepta Therapeutics) for ...
Posted by Michael Wonder on 09 Jun 2023
FDA accepts biologics license applications for exagamglogene autotemcel (exa-cel) for severe sickle cell disease and transfusion-dependent beta thalassaemia
8 June 2023 - FDA grants priority review for severe sickle cell disease and standard review for transfusion-dependent beta thalassemia. ...
Posted by Michael Wonder on 08 Jun 2023
Rocket Pharmaceuticals receives FDA fast track and orphan drug designations for RP-A601 gene therapy for PKP2 arrhythmogenic cardiomyopathy
8 June 2023 - Rocket Pharmaceuticals today announced that the US FDA has granted fast track and orphan drug designations ...
Posted by Michael Wonder on 01 Jun 2023
Arcturus Therapeutics receives US FDA fast track designation for ARCT-810, mRNA therapeutic candidate for ornithine transcarbamylase deficiency
1 June 2023 - Arcturus Therapeutics today announced that the US FDA has granted fast track designation to ARCT-810, the Company’s ...
Posted by Michael Wonder on 24 May 2023
Sarepta Therapeutics announces update on regulatory review of SRP-9001
24 May 2023 - New regulatory action date is 22 June 2023. ...
Posted by Michael Wonder on 23 May 2023
RegenxBio receives FDA regenerative medicine advanced therapy designation for RGX-121 gene therapy for Hunter syndrome
23 May 2023 - CAMPSIITE trial is enrolling mucopolysaccharidosis type II patients as part of a pivotal program that incorporates ...