Blog
RSS FeedPosted 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. ...
Posted by Michael Wonder on 18 Sep 2022
bluebird bio receives FDA accelerated approval for Skysona gene therapy for early, active cerebral adrenoleukodystrophy
16 September 2022 - Skysona is the first FDA approved therapy shown to slow the progression of neurologic dysfunction in boys ...
Posted by Michael Wonder on 15 Sep 2022
Aro Biotherapeutics receives FDA rare paediatric drug designation for ABX1100 for the treatment of Pompe disease
15 September 2022 - Aro Biotherapeutics today announced that the US FDA granted rare paediatric drug designation for ABX1100, an investigational ...
Posted by Michael Wonder on 08 Sep 2022
Silence Therapeutics announces FDA fast track designation for SLN124, a novel investigational siRNA therapy for the treatment of polycythemia vera
8 September 2022 - Designation provides potential for expedited drug development path. ...
Posted by Michael Wonder on 25 Aug 2022
First gene therapy for adults with severe haemophilia A, BioMarin's Roctavian (valoctocogene roxaparvovec), approved by European Commission
24 August 2022 - Maintains orphan drug designation in the EU providing 10 years of market exclusivity. ...
Posted by Michael Wonder on 23 Aug 2022
iECURE receives FDA rare paediatric disease designation for GTP-506, an investigational gene editing product candidate for the treatment of ornithine transcarbamylase deficiency
23 August 2022 - GTP-506, a potential single-dose gene editing therapy designed to restore metabolic function in patients suffering with OTC ...
Posted by Michael Wonder on 23 Jul 2022
Alnylam receives positive CHMP opinion for vutrisiran for the treatment of hereditary transthyretin-mediated amyloidosis in adult patients with stage 1 or stage 2 polyneuropathy
22 July 2022 - Positive opinion based on HELIOS-A Phase 3 study. ...
Posted by Michael Wonder on 04 Jul 2022
Report on 'Cell, Gene and Tissue Regulatory Framework in Australia: Stakeholder Perspectives' - TGA response
4 July 2022 - n November 2021 the TGA commissioned MTP Connect to conduct a stakeholder review of the regulatory ...
Posted by Michael Wonder on 28 Jun 2022
Adverum Biotechnologies granted Priority Medicines (PRIME) designation by EMA for ADVM-022 in wet AMD
24 June 2022 - Adverum Biotechnologies today announced that the EMA has granted ADVM-022 Priority Medicines (PRIME) designation for the treatment ...
Posted by Michael Wonder on 26 Jun 2022
First gene therapy to treat severe haemophilia A
24 June 2022 - EMA has recommended granting a conditional marketing authorisation in the European Union (EU) for Roctavian (valoctocogene roxaparvovec) ...
Posted by Michael Wonder on 14 Jun 2022
Alnylam announces FDA approval of Amvuttra (vutrisiran), an RNAi therapeutic for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults
13 June 2022 - First and only FDA approved treatment demonstrating reversal in neuropathy impairment with subcutaneous administration once every three ...
Posted by Michael Wonder on 24 May 2022
FDA accepts CSL Behring's biologics license application for etranacogene dezaparvovec for priority review
24 May 2022 - If approved, etranacogene dezaparvovec would be the first gene therapy option for people living with haemophilia B. ...
Posted by Michael Wonder on 21 May 2022
PTC Therapeutics receives positive CHMP opinion for Upstaza for the treatment of AADC deficiency
20 May 2022 - First ever gene therapy directly administered into the brain. ...