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RSS FeedPosted by Michael Wonder on 03 Sep 2024
Obsidian Therapeutics receives FDA regenerative medicine advanced therapy designation for OBX-115 for the treatment of advanced melanoma
3 September 2024 - Obsidian Therapeutics today announced that the US FDA has granted regenerative medicine advanced therapy designation to ...
Posted by Michael Wonder on 02 Sep 2024
CAMP4 Therapeutics secures rare paediatric disease designation for CMP-CPS-001 for the treatment of urea cycle disorders
27 August 2024 - CAMP4 Therapeutics today announced the US FDA has granted rare paediatric disease designation to the Company’s lead ...
Posted by Michael Wonder on 01 Sep 2024
Ipsen announces sale of priority review voucher for $158 million
27 August 2024 - Ipsen has entered into an agreement to sell its rare paediatric disease priority review voucher to ...
Posted by Michael Wonder on 22 Aug 2024
Opus Genetics receives rare paediatric disease designation from the US FDA for ocular gene therapy OPGx-LCA5 to treat rare inherited retinal disease LCA5
20 August 2024 - Opus Genetics today announced the US FDA has granted rare paediatric disease designation for its ocular gene ...
Posted by Michael Wonder on 15 Aug 2024
Imfinzi granted priority review and breakthrough therapy designation for patients with limited-stage small cell lung cancer in the US
15 August 2024 - Based on ADRIATIC Phase 3 trial which demonstrated statistically significant and clinically meaningful overall survival and ...
Posted by Michael Wonder on 13 Aug 2024
Wave Life Sciences receives FDA rare paediatric disease designation for WVE-N531 for the treatment of Duchenne muscular dystrophy
12 August 2024 - Wave Life Sciences today announced that the US FDA has granted rare paediatric disease designation to WVE-N531 ...
Posted by Michael Wonder on 12 Aug 2024
Actio Biosciences receives orphan drug and rare paediatric disease designations for the treatment of Charcot-Marie-Tooth disease 2C
8 August 2024 - Actio Biosciences today announced the US FDA has granted both orphan drug designation and rare paediatric disease ...
Posted by Michael Wonder on 11 Aug 2024
Satellos receives rare paediatric disease designation from the US FDA for SAT-3247 for the treatment of Duchenne muscular dystrophy
8 August 2024 - Satellos Bioscience announced today that the US FDA has granted rare paediatric disease designation to SAT-3247 ...
Posted by Michael Wonder on 08 Aug 2024
Neurogene announces RMAT designation for NGN-401 investigational gene therapy for Rett syndrome
7 August 2024 - Designation based on preliminary clinical evidence from ongoing NGN-401 clinical trial that shows potential to address unmet ...
Posted by Michael Wonder on 07 Aug 2024
Meta Pharmaceuticals announces FDA grants rare paediatric disease designation to META-001-PH for the treatment of primary hyperoxaluria
5 August 2024 - META Pharmaceuticals Inc. announced that the US FDA has granted rare paediatric disease designation to its investigational ...
Posted by Michael Wonder on 03 Aug 2024
FDA has accepted a BLA for bentracimab, the first and only ticagrelor reversal agent, for filing and priority review
2 August 2024 - SFJ Pharmaceuticals, sponsor of the bentracimab biologics license application, and SERB Pharmaceuticals (SERB), who acquired exclusive ...
Posted by Michael Wonder on 02 Aug 2024
Adverum Biotechnologies announces FDA regenerative medicine advanced therapy designation granted for ixo-vec for the treatment of wet AMD
1 August 2024 - Adverum Biotechnologies today announced that the US FDA has granted regenerative medicine advanced therapy designation for ...
Posted by Michael Wonder on 01 Aug 2024
Introducing a new way of prioritising our guidance topics
30 July 2024 - NICE's deputy chief executive, Jonathan Benger, talks about our new approach to prioritisation. ...
Posted by Michael Wonder on 30 Jul 2024
AffyImmune receives FDA regenerative medicine advanced therapy designation for AIC100 in recurrent anaplastic thyroid cancer
23 July 2024 - RMAT designation follows FDA’s review of available preliminary clinical evidence that AIC100 has the potential to address ...
Posted by Michael Wonder on 30 Jul 2024
Novartis Scemblix granted FDA priority review for the treatment of adults with newly diagnosed CML
29 July 2024 - Priority review based on ASC4FIRST Phase 3 study with Scemblix data first to show significantly improved molecular ...