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RSS FeedPosted 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 ...
Posted by Michael Wonder on 14 Nov 2024
PTC Therapeutics announces FDA approval of AADC deficiency gene therapy
13 November 2024 - PTC Therapeutics announced today the US FDA accelerated approval of its gene therapy for the treatment of ...
Posted by Michael Wonder on 12 Nov 2024
Abeona Therapeutics announces FDA acceptance of BLA resubmission of prademagene zamikeracel for the treatment of recessive dystrophic epidermolysis bullosa
12 November 2024 - Abeona Therapeutics today announced that the US FDA has accepted for review Abeona’s resubmission of its ...
Posted by Michael Wonder on 12 Nov 2024
Genascence granted FDA fast track designation for GNSC-001 in patients with osteoarthritis of the knee
12 November 2024 - GNSC-001 is designed to offer long-term, sustained inhibition of IL-1 following a single injection into the affected ...
Posted by Michael Wonder on 31 Oct 2024
Abeona Therapeutics completes Pz-cel biologics license application resubmission to US FDA
29 October 2024 - Abeona Therapeutics today announced that the Company has resubmitted its biologics license application to the US FDA ...
Posted 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 ...