Blog
RSS FeedPosted by Michael Wonder on 30 Oct 2024
Eplontersen for the treatment of patients with hereditary transthyretin related amyloidosis
29 October 2024 - NICE has published final drat guidance on the use of eplontersen for the treatment of patients ...
Posted by Michael Wonder on 22 Oct 2024
ICER publishes final evidence report on treatments for transthyretin amyloid cardiomyopathy
21 October 2024 - Independent appraisal committee voted that current evidence is adequate to demonstrate superior net health benefits for ...
Posted by Michael Wonder on 21 Oct 2024
Wainzua (eplontersen) recommended for approval in the EU by CHMP for the treatment of adult patients with polyneuropathy associated with hereditary transthyretin-mediated amyloidosis
21 October 2024 - Recommendation based on NEURO-TTRansform Phase 3 results showing Wainzua demonstrated consistent and sustained benefit improving neuropathy ...
Posted by Michael Wonder on 17 Oct 2024
Alnylam submits regulatory application to the EMA for vutrisiran for the treatment of ATTR amyloidosis with cardiomyopathy
16 October 2024 - Type II variation submission based on the positive HELIOS-B Phase 3 trial in which vutrisiran significantly reduced ...
Posted by Michael Wonder on 10 Oct 2024
Alnylam submits supplemental new drug application to the US FDA for vutrisiran for the treatment of transthyretin amyloidosis with cardiomyopathy
9 October 2024 - Priority review voucher utilised to accelerate review period. ...
Posted by Michael Wonder on 26 Sep 2024
Health Canada grants marketing authorisation of first CRISPR/Cas9 gene edited therapy, Casgevy (exagamglogene autotemcel), for the treatment of sickle cell disease and transfusion-dependent beta thalassaemia
25 September 2024 - First regulatory authorisation of a CRISPR-based gene-editing therapy in Canada. ...
Posted by Michael Wonder on 17 Sep 2024
ICER to assess treatment for retinitis pigmentosa
17 September 2024 - Report will be subject of New England CEPAC meeting in April 2025; draft scoping document open ...
Posted by Michael Wonder on 12 Sep 2024
Ilya Pharma receives rare paediatric disease designation from the US FDA for emilimogene sigulactibac for the treatment of skin wounds in SAVI patients
11 September 2024 - Ilya Pharma today announced that it has received rare paediatric disease designation by the US FDA. ...
Posted by Michael Wonder on 11 Sep 2024
Exagamglogene autotemcel for the treatment of patients 12 years of age and older with transfusion-dependent beta-thalassaemia
11 September 2024 - NICE has published final evidence-based recommendations on the use of exagamglogene autotemcel (Casgevy) for the treatment ...
Posted by Michael Wonder on 10 Sep 2024
BridgeBio receives FDA’s regenerative medicine advanced therapy designation for BBP-812 Canavan disease gene therapy program
10 September 2024 - Receipt of RMAT designation is based on preliminary clinical evidence from the CANaspire Phase 1/2 clinical trial, ...
Posted by Michael Wonder on 08 Sep 2024
YolTech Therapeutics receives US FDA rare paediatric disease designation for YOLT-203 in treating primary hyperoxaluria type 1
4 September 2024 - YolTech Therapeutics today announced that the US FDA has granted rare paediatric disease designation to YOLT-203 for ...
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 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 09 Aug 2024
BioMarin announces updated strategy for Roctavian to focus on US, Germany and Italy
5 August 2024 - Strategic focus will reduce operating expenses with the goal of achieving Roctavian profitability by end of 2025. ...
Posted by Michael Wonder on 08 Aug 2024
World’s first gene editing therapy for blood disorder to be available to hundreds of patients in England
8 August 2024 - Patients in England with severe beta-thalassaemia will be amongst the first in Europe to benefit from one-time ...