Blog
RSS FeedPosted by Michael Wonder on 13 Aug 2019
Memo to the Novartis CEO: when in doubt, give the FDA a heads-up
13 August 2019 - For someone who has made a point of trying to steer Novartis away from scandal, Vas ...
Posted by Michael Wonder on 12 Aug 2019
Democratic senators urge FDA to take action against drug company that hid faulty data
9 August 2019 - A group of Democratic senators is urging the Food and Drug Administration (FDA) to take action ...
Posted by Michael Wonder on 06 Aug 2019
Novartis stands behind Zolgensma (onasemnogene abeparvovec-xioi) for the treatment of children less than 2 years of age with spinal muscular atrophy
6 August 2019 - Today the FDA released a statement addressing data integrity issues with the Biologics License Application (BLA) for ...
Posted by Michael Wonder on 06 Aug 2019
Novartis hid manipulated data while seeking approval for $2.1 million treatment
6 August 2019 - The failure to report the issue has not put patients at risk, the F.D.A. said, but the ...
Posted by Michael Wonder on 06 Aug 2019
Statement on data accuracy issues with recently approved gene therapy
6 August 2019 - As a public health agency, we believe that it is critical to facilitate the development of ...
Posted by Michael Wonder on 05 Aug 2019
Alnylam announces U.S. FDA granted priority review of the givosiran new drug application for the treatment of acute hepatic porphyria
5 August 2019 - PDUFA date set for 4 February 2020. ...
Posted by Michael Wonder on 17 Jul 2019
LogicBio Therapeutics receives rare paediatric disease designation for LB-001 for the treatment of methylmalonic acidaemia
16 July 2019 - LogicBio Therapeutics today announced the U.S. FDA has granted rare paediatric disease designation to LB-001, a ...
Posted by Michael Wonder on 16 Jul 2019
Dicerna receives breakthrough therapy designation for DCR-PHXC for treatment of primary hyperoxaluria type 1
15 July 2019 - FDA recognises primary hyperoxaluria Types 2 and 3 as meeting criteria for a serious or life-threatening disease ...
Posted by Michael Wonder on 03 Jul 2019
Some insurers are pushing back on the high price for a Novartis gene therapy
3 July 2019 - Barely a month after Novartis won U.S. regulatory approval for its gene therapy — the most ...
Posted by Michael Wonder on 02 Jul 2019
Innovation prizes to support cell and gene therapy
2 July 2019 - Personalised cell and gene therapy represent a fundamental therapeutic revolution. ...
Posted by Michael Wonder on 25 Jun 2019
Krystal Biotech receives regenerative medicine advanced therapy designation from FDA for KB103
24 June 2019 - RMAT designation granted to KB103 based on positive interim data from GEM-1 and GEM-2 study. ...
Posted by Michael Wonder on 19 Jun 2019
Abeona Therapeutics receives FDA fast track designation for ABO-202 AAV9 gene therapy in CLN1 disease
18 June 2019 - Abeona Therapeutics today announced that the U.S. Food and FDA has granted fast track designation to its ...
Posted by Michael Wonder on 06 Jun 2019
Alnylam completes rolling submission of new drug application to U.S. FDA for givosiran for the treatment of acute hepatic porphyria
5 June 2019 - First potential treatment demonstrating substantial reduction in the frequency of porphyria attacks. ...
Posted by Michael Wonder on 30 May 2019
Fibrocell receives FDA regenerative medicine advanced therapy designation for FCX-007 gene therapy for the treatment of RDEB
29 May 2019 - Fibrocell Science today announced that the U.S. FDA has granted the regenerative medicine advanced therapy designation to ...
Posted by Michael Wonder on 25 May 2019
RegenxBio announces first FDA approval of a gene therapy based on its proprietary NAV technology platform
24 May 2019 - FDA approval of Novartis' Zolgensma for the treatment of paediatric patients with SMA marks the first regulatory ...