Blog
RSS FeedPosted 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
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 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 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 13 Oct 2022
FDA accepts BioMarin's biologics license application for valoctocogene roxaparvovec AAV gene therapy for adults with severe haemophilia A
12 October 2022 - PDUFA target action date is 31 March 2023 ...
Posted by Michael Wonder on 07 Oct 2022
Alnylam announces FDA approval of supplemental new drug application for Oxlumo (lumasiran) in advanced primary hyperoxaluria type 1
6 October 2022 - Approval is based on positive efficacy and safety results of the ILLUMINATE-C Phase 3 study of Oxlumo ...
Posted by Michael Wonder on 29 Sep 2022
BioMarin resubmits biologics license application for valoctocogene roxaparvovec AAV gene therapy for severe haemophilia A to the FDA
29 September 2022 - BLA includes substantial body of data from pivotal Phase 3 and on-going Phase 1/2 studies. ...
Posted by Michael Wonder on 21 Sep 2022
Alnylam receives approval in Europe for Amvuttra (vutrisiran) for the treatment of hereditary transthyretin-mediated amyloidosis in adult patients with stage 1 or stage 2 polyneuropathy
20 September 2022 - Amvuttra demonstrated halting or reversal in neuropathy impairment with subcutaneous administration once every three months. ...
Posted by Michael Wonder on 20 Sep 2022
AvroBio receives rare paediatric disease designation from US FDA for first gene therapy in development for cystinosis
20 September 2022 - AVR-RD-04 has previously received orphan drug designation from FDA and EMA. ...