Blog
RSS FeedPosted by Michael Wonder on 31 Oct 2023
US FDA awards both rare paediatric disease and orphan drug designations to HG204, a CRISPR RNA editing therapy, for the treatment of MECP2 duplication syndrome
31 October 2023 - HG204 is the world first CRISPR RNA-editing therapy for the treatment of MECP2 duplication syndrome granted ...
Posted by Michael Wonder on 30 Oct 2023
ICER publishes final evidence report on gene therapy for metachromatic leukodystrophy
30 October 2023 - Independent appraisal committee voted that across all patient sub-populations, arsa-cel demonstrated a net health benefit when ...
Posted by Michael Wonder on 27 Oct 2023
Health Canada authorises CSL's Hemgenix (etranacogene dezaparvovec) as first gene therapy for haemophilia B
26 October 2023 - CSL's Hemgenix is a one time, single dose treatment for adults with haemophilia B who require ...
Posted by Michael Wonder on 23 Oct 2023
Life changing gene therapy for babies with spinal muscular atrophy
23 October 2023 - The Australian Government is giving more families access to life changing gene therapy for spinal muscular atrophy ...
Posted by Michael Wonder on 19 Oct 2023
Health economic assessment of Spinraza for type 5q spinal muscular atrophy
18 October 2023 - TLV has produced a health economic knowledge base for the regions for Spinraza (nusinersen sodium) for the ...
Posted by Michael Wonder on 14 Oct 2023
Intellia Therapeutics receives Priority Medicines (PRIME) designation from the EMA for NTLA-2002, an investigational in vivo CRISPR genome editing treatment for hereditary angioedema
13 October 2023 - Intellia Therapeutics today announced that the EMA has granted Priority Medicine (PRIME) designation to NTLA-2002 for ...
Posted by Michael Wonder on 12 Oct 2023
Health economic assessment of the gene therapy Hemgenix in severe and moderate haemophilia B
11 October 2023 - TLV has developed a health economic knowledge base for the regions for the gene therapy Hemgenix (etranacogene ...
Posted by Michael Wonder on 09 Oct 2023
Alnylam announces receipt of complete response letter from US FDA for supplemental new drug application for patisiran for the treatment of the cardiomyopathy of ATTR amyloidosis
9 October 2023 - FDA cites insufficient evidence of clinical meaningfulness. ...
Posted by Michael Wonder on 03 Oct 2023
FDA approves Rivfloza for children ≥9 years old and adults living with primary hyperoxaluria type 1, a rare genetic condition
2 October 2023 - Novo Nordisk announced today that the US FDA has approved Rivfloza (nedosiran) injection 80 mg, 128 mg, ...
Posted by Michael Wonder on 03 Oct 2023
Rocket Pharmaceuticals announces FDA acceptance of biologics license application with priority review for RP-L201 (marnetegragene autotemcel) for the treatment of severe leukocyte adhesion deficiency-I
2 October 2023 - PDUFA target action date is 31 March 2024. ...
Posted by Michael Wonder on 19 Sep 2023
Orchard Therapeutics announces acceptance of biologics license application for OTL-200 in MLD and receives priority review
18 September 2023 - PDUFA date set for 18 March 2024. ...
Posted by Michael Wonder on 14 Sep 2023
ICER publishes evidence report on gene therapy for metachromatic leukodystrophy
14 September 2023 - Using weighted analyses across all patient subpopulations, atidarsagene autotemcel would achieve common thresholds for cost effectiveness if priced ...
Posted by Michael Wonder on 13 Sep 2023
First bladder cancer patient dosed with commercially available intravesical gene therapy Adstiladrin (nadofaragene firadenovec-vncg)
12 September 2023 - Ferring launches ABLE-41 Real World Evidence Study, a patient registry to explore early use, experiences and outcomes ...
Posted by Michael Wonder on 30 Aug 2023
Bloomsbury Genetic Therapies receives rare paediatric disease designation from the US FDA for BGT-INAD for the treatment of infantile neuroaxonal dystrophy
29 August 2023 - Bloomsbury Genetic Therapies Limited announced today that the US FDA has granted rare paediatric disease designation for ...
Posted by Michael Wonder on 24 Aug 2023
Taysha Gene Therapies announces fast track designation granted by US FDA for TSHA-102 in Rett syndrome
24 August 2023 - TSHA-102 has also received orphan drug and rare paediatric disease designations from the US FDA and ...