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RSS FeedPosted by Michael Wonder on 07 Oct 2023
Zatolmilast, an investigational treatment for Fragile X syndrome, receives rare paediatric disease designation from the US FDA
27 September 2023 - Shionogi announced the US FDA has granted Tetra Therapeutics, a Shionogi Group Company, rare paediatric disease designation ...
Posted by Michael Wonder on 28 Sep 2023
FDA approves bosutinib for paediatric patients with chronic myelogenous leukaemia
26 September 2023 - Today, the FDA approved bosutinib (Bosulif, Pfizer) for paediatric patients 1 year of age and older ...
Posted by Michael Wonder on 26 Sep 2023
Dupixent (dupilimab) sBLA for treatment of eosinophilic oesophagitis in children aged 1 to 11 accepted for FDA priority review
26 September 2023 - If approved, Dupixent would be the first and only treatment in the US indicated for children aged ...
Posted by Michael Wonder on 19 Sep 2023
Altuviiio supplemental biologics license application based on positive final results from Phase 3 XTEND-Kids study accepted by FDA
12 September 2023 - Final results from XTEND-Kids study were submitted for review, potentially expanding on the interim data included in ...
Posted by Michael Wonder on 18 Sep 2023
BioMarin receives positive CHMP opinion in Europe to expand use of Voxzogo (vosoritide) to treat children aged 4 months and older with achondroplasia
15 September 2023 - BioMarin today announced that the EMA's CHMP has adopted a positive opinion recommending marketing authorisation to expand ...
Posted by Michael Wonder on 16 Sep 2023
Vertex receives CHMP positive opinion for Kaftrio (ivacaftor/tezacaftor/elexacaftor) in combination with ivacaftor for children with cystic fibrosis ages 2 through 5
15 September 2023 - If approved, more than 1,200 children would be newly eligible for a medicine that could treat ...
Posted by Michael Wonder on 10 Sep 2023
European Commission expands Merck’s Ervebo [Ebola Zaire vaccine, live] indication to include children 1 year of age and older
7 September 2023 - Milestone signifies on-going effort to help prepare for outbreaks of Zaire ebolavirus. ...
Posted by Michael Wonder on 30 Aug 2023
European Commission approves Roche’s Evrysdi for babies under two months old with spinal muscular atrophy
29 August 2023 - Approval is based on interim data from ongoing RAINBOWFISH trial showing majority of Evrysdi-treated babies were able ...
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 15 Aug 2023
Lactiga receives FDA rare paediatric disease designation for groundbreaking mucosal immunoglobulin therapy for common variable immunodeficiency disease
14 August 2023 - LCTG-001 on course to be the first FDA approved biologic from human milk. ...
Posted by Michael Wonder on 14 Aug 2023
In the case of a devastating disease, the FDA weighs an experimental drug’s muddled data and a desperate need
14 August 2023 - Emma Albee knows the experimental drug she takes is not a cure. It won’t allow her ...
Posted by Michael Wonder on 10 Aug 2023
US FDA accepts Astellas' sNDA for Cresemba (isavuconazonium sulphate) in children
10 August 2023 - Astellas announced that the US FDA has accepted the company's supplemental new drug application for Cresemba (isavuconazonium ...
Posted by Michael Wonder on 07 Aug 2023
HuidaGene Therapeutics receives FDA rare paediatric disease designation for HG004 to treat inherited blindness
7 August 2023 - HG004 is a one time, direct to RPE treatment of inherited retinal disease caused by mutations in ...
Posted by Michael Wonder on 04 Aug 2023
Mesoblast receives complete response from US FDA for biologics license application for steroid-refractory acute graft versus host disease in children
4 August 2023 - Mesoblast Limited today announced that the US FDA has provided a complete response to its biologics license ...
Posted by Michael Wonder on 03 Aug 2023
Aardvark Therapeutics announces receipt of FDA rare paediatric disease designation for Prader-Willi syndrome
3 August 2023 - Aardvark's Phase 2 trial of oral ARD-101 in young adults with Prader-Willi syndrome is now open ...