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RSS FeedPosted by Michael Wonder on 18 Jan 2025
Arrowhead Pharmaceuticals announces acceptance of new drug application by US FDA of plozasiran for the treatment of familial chylomicronaemia syndrome
17 January 2025 - The new drug application is based on positive results from the Phase 3 PALISADE study. ...
Posted by Michael Wonder on 14 Jan 2025
Adaptimmune announces US FDA breakthrough therapy designation granted to letetresgene autoleucel for treatment of myxoid/round cell liposarcoma
13 January 2025 - Adaptimmune Therapeutics today announced that letetresgene autoleucel has been granted breakthrough therapy designation by the US ...
Posted by Michael Wonder on 10 Jan 2025
ViGeneron announces FDA rare paediatric disease designation for VG901
8 January 2025 - ViGeneron today announced two important milestones for its novel gene therapy candidate VG901, to treat patients ...
Posted by Michael Wonder on 07 Jan 2025
ICER to assess treatments for spinal muscular atrophy
6 January 2025 - Report will be subject of Midwest CEPAC meeting in August 2025; draft scoping document open to ...
Posted by Michael Wonder on 27 Dec 2024
Alnylam Canada signs letter of intent from pan-Canadian Pharmaceutical Alliance for the public reimbursement of Amvuttra for the treatment of hereditary transthyretin-mediated amyloidosis in adults
18 October 2024 - One step closer to achieving public reimbursement of Amvuttra for hereditary transthyretin-mediated amyloidosis. patients in Canada ...
Posted by Michael Wonder on 21 Dec 2024
Ultragenyx submits biologics license application to the US FDA for UX111 AAV gene therapy for the treatment of Sanfilippo syndrome type A
19 December 2024 - If approved, UX111 would be the first approved therapy in the US for Sanfilippo syndrome type ...
Posted by Michael Wonder on 13 Dec 2024
Stoke Therapeutics receives FDA breakthrough therapy designation for zorevunersen for the treatment of Dravet syndrome
4 December 2024 - Supported by evidence from clinical studies that indicate that zorevunersen may demonstrate substantial improvement over available therapies. ...
Posted by Michael Wonder on 11 Dec 2024
Krystal Biotech provides update on EMA’s on-going regulatory review of B-VEC for dystrophic epidermolysis bullosa
9 December 2024 - CHMP opinion now expected in Q1, 2025. ...
Posted by Michael Wonder on 10 Dec 2024
MeiraGTx granted FDA regenerative medicine advanced therapy designation for AAV2-hAQP1 for the treatment of grade 2/3 radiation-induced xerostomia
9 December 2024 - MeiraGTx today announced that the US FDA has granted regenerative medicine advanced therapy designation to AAV2-hAQP1 for ...
Posted by Michael Wonder on 07 Dec 2024
Biden-Harris Administration takes next steps to increase access to sickle cell disease treatments
4 December 2024 - Today, the Biden-Harris Administration announced that two drug manufacturers with FDA approved gene therapies for sickle ...
Posted by Michael Wonder on 03 Dec 2024
Intellia Therapeutics announces FDA regenerative medicine advanced therapy designation granted to nexiguran ziclumeran for the treatment of hereditary transthyretin amyloidosis with polyneuropathy
25 November 2024 - Intellia Therapeutics today announced that the US FDA has granted regenerative medicine advanced therapy designation to nexiguran ...
Posted by Michael Wonder on 29 Nov 2024
Replimune receives breakthrough therapy designation for RP1 and submits RP1 biologics license application to the FDA under the accelerated approval pathway
21 November 2024 - Replimune today announced that it has submitted a biologics license application to the FDA for RP1 (vusolimogene ...
Posted by Michael Wonder on 21 Nov 2024
Arrowhead Pharmaceuticals submits new drug application to US FDA for plozasiran for the treatment of familial chylomicronemia syndrome
18 November 2024 - The new drug application is based on positive results from the Phase 3 PALISADE study. ...
Posted by Michael Wonder on 16 Nov 2024
AlveoGene receives rare paediatric disease designation from FDA for AVG-002, its novel, inhaled gene therapy for lethal neonatal surfactant protein B deficiency
15 November 2024 - AlveoGene announces that it has been granted a rare paediatric disease designation by the US FDA ...
Posted by Michael Wonder on 14 Nov 2024
Qalsody: HAS update
8 November 2024 - On 10 October, the High Authority for Health (HAS) issued a negative decision on the request for ...