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RSS FeedPosted by Michael Wonder on 24 Dec 2019
BioMarin submits biologics license application to U.S. FDA for valoctocogene roxaparvovec to treat haemophilia A
23 December 2019 - First marketing application submission in U.S. for gene therapy directed at any type of haemophilia. ...
Posted by Michael Wonder on 14 Dec 2019
FDA grants accelerated approval to first targeted treatment for rare Duchenne muscular dystrophy mutation
12 December 2019 - The U.S. FDA today granted accelerated approval to Vyondys 53 (golodirsen) injection to treat Duchenne muscular dystrophy ...
Posted by Michael Wonder on 13 Dec 2019
Sarepta Therapeutics announces FDA approval of Vyondys (golodirsen) injection for the treatment of Duchenne muscular dystrophy in patients amenable to skipping exon 53
12 December 2019 - Vyondys 53 is Sarepta’s second RNA exon-skipping treatment for DMD approved in the U.S. ...
Posted by Michael Wonder on 25 Nov 2019
FDA grants priority review to Roche’s risdiplam for spinal muscular atrophy
25 November 2019 - Filing submission includes 12-month data from pivotal FIREFISH and SUNFISH trials in a broad population of people ...
Posted by Michael Wonder on 21 Nov 2019
Drug for ultra rare disease comes with ultra high price tag
21 November 2019 - Givlaari’s net price will be $442,000 after discounts included. ...
Posted by Michael Wonder on 20 Nov 2019
FDA approves first treatment for inherited rare disease
20 November 2019 - Today, the U.S. FDA granted approval to Givlaari (givosiran) for the treatment of adult patients with acute ...
Posted by Michael Wonder on 06 Nov 2019
Novartis says delayed telling FDA of Zolgensma concern due to 'mistake'
2 November 2019 - Novartis scientists learnt months ago about safety concerns surrounding its gene therapy Zolgensma but delayed telling the ...
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 22 Oct 2019
Moderna receives FDA fast track designation for propionic acidemia program (mRNA-3927)
22 October 2019 - mRNA-3927 is Moderna’s second rare disease program to receive fast track designation. ...
Posted by Michael Wonder on 24 Sep 2019
Novartis unit says FDA wasn’t told of data manipulation sooner because it was ‘highly complex’
25 September 2019 - If you were wondering why Novartis waited more than three months to notify the Food and ...
Posted by Michael Wonder on 24 Sep 2019
FDA grants paediatric disease designation for Mateon's OT101
23 September 2019 - For treatment of diffuse intrinsic pontine glioma, a difficult to treat form of childhood brain tumour. ...
Posted by Michael Wonder on 15 Sep 2019
The startup that manipulated data to get a miracle drug to market
14 September 2019 - AveXis had a revolutionary gene therapy for babies with a deadly disease, and sold to Novartis for ...
Posted by Michael Wonder on 09 Sep 2019
Reeling from scandal, Novartis vows to voluntarily release info on manipulated data in the future
9 September 2019 - Seeking to appease angry regulators, Novartis committed to notifying the FDA within five business days of ...
Posted by Michael Wonder on 04 Sep 2019
Magenta Therapeutics announces FDA regenerative medicine advanced therapy designation granted to MGTA-456 for the treatment of inherited metabolic disorders
4 September 2019 - Magenta Therapeutics today announced that the U.S. FDA granted regenerative medicine advanced therapy designation for MGTA-456, ...
Posted by Michael Wonder on 27 Aug 2019
An FDA report reveals the agency believed Novartis data problems were resolved months ago
27 August 2019 - Shortly before serious data problems surfaced in mid-March at a troubled Novartis unit, a Food and ...