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RSS FeedPosted by Michael Wonder on 03 Dec 2022
Orchard Therapeutics announces Swissmedic validation of the marketing authorisation application for Libmeldy (atidarsagene autotemcel)
1 December 2022 - Orchard Therapeutics today announced its marketing authorisation application for Libmeldy (atidarsagene autotemcel) has been accepted for ...
Posted by Michael Wonder on 30 Nov 2022
How CSL priced the world’s most expensive drug
1 December 2022 - When blood products giant CSL announced last week it had been given US regulatory approval for ...
Posted by Michael Wonder on 28 Nov 2022
Sarepta Therapeutics announces that US FDA has accepted for filing and granted priority review for the biologics license application for SRP-9001, Sarepta’s gene therapy for the treatment of ambulant individuals with Duchenne muscular dystrophy
28 November 2022 - Regulatory action date of 29 May 2023. ...
Posted by Michael Wonder on 23 Nov 2022
ICER to assess gene therapies for sickle cell disease
23 November 2022 - Report will be subject of CTAF meeting in June 2023; draft scoping document open to public ...
Posted by Michael Wonder on 23 Nov 2022
BioMarin announces advancements in FDA review of Roctavian (valoctocogene roxaparvovec) for adults with severe haemophilia A
23 November 2022 - FDA no longer plans to hold an advisory committee meeting, as previously planned, to discuss the biologics ...
Posted by Michael Wonder on 23 Nov 2022
CSL lands world first gene therapy with $5.3 million haemophilia treatment
23 November 2022 - Australian biotech giant CSL will bring the first gene therapy for haemophilia B to market in ...
Posted by Michael Wonder on 23 Nov 2022
FDA approves first gene therapy to treat adults with haemophilia B
22 November 2022 - Today, the US FDA approved Hemgenix (etranacogene dezaparvovec), an adeno-associated virus vector-based gene therapy for the treatment ...
Posted by Michael Wonder on 08 Nov 2022
BioMarin announces incremental progress on biologics license application review for valoctocogene roxaparvovec AAV gene therapy for adults with severe haemophilia A program
7 November 2022 - FDA requests submission of upcoming 3 year data analysis from Phase 3 GENEr8-1 trial. ...
Posted by Michael Wonder on 07 Nov 2022
Sensorion announces US FDA grants rare paediatric disease designation to OTOF-GT for the treatment of otoferlin gene-mediated hearing loss
7 November 2022 - OTOF-GT targets the restoration of hearing in people living with otoferlin deficiency. ...
Posted by Michael Wonder on 05 Nov 2022
FDA finalises umbrella trial guidance for cell and gene therapies
4 November 2022 - The US FDA laid out its recommendations for sponsors to study multiple versions of a cellular or ...
Posted by Michael Wonder on 02 Nov 2022
ICER publishes evidence report on gene therapies for haemophilia A and B
2 November 2022 - Duration of benefit with gene therapies and risks for rare side effects remain major uncertainties. ...
Posted by Michael Wonder on 27 Oct 2022
AvroBio receives rare paediatric disease designation from the US FDA for first in class gene therapy for Gaucher disease
27 October 2022 - AVR-RD-02 has previously received fast track status from FDA, orphan drug designation in the US and EU, ...
Posted by Michael Wonder on 18 Oct 2022
AvroBio granted ILAP designation from UK MHRA for first in class gene therapy for Gaucher disease
18 October 2022 - ILAP intended to accelerate regulatory review process and facilitate patient access in UK for seriously debilitating and ...
Posted by Michael Wonder on 17 Oct 2022
Biogen announces FDA’s 3 month extension of review period for the new drug application for tofersen
17 October 2022 - The new Prescription Drug User Fee Act action date set by the FDA is 25 April ...
Posted by Michael Wonder on 17 Oct 2022
Gene therapy Luxturna now reimbursed in Quebec for people with previously untreatable inherited vision loss
17 October 2022 - Quebec leads the way as the first province to reimburse Luxturna (voretigene neparvovec) for previously untreatable inherited ...