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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 31 Oct 2023
Day One announces FDA acceptance of NDA and priority review for tovorafenib in relapsed or progressive paediatric low grade glioma
30 October 2024 - Priority review granted with PDUFA target action date of 30 April 2024. ...
Posted by Michael Wonder on 22 Oct 2023
US Food and Drug Administration approves BioMarin's Voxzogo (vosoritide) for children under 5 years with achondroplasia
20 October 2023 - Expanded indication in the US now includes children of all ages with achondroplasia. ...
Posted by Michael Wonder on 20 Oct 2023
FDA expands paediatric indication for entrectinib and approves new pellet formulation
20 October 2023 - Today, the FDA granted accelerated approval to entrectinib (Rozlytrek, Genentech) for paediatric patients older than 1 ...
Posted by Michael Wonder on 17 Oct 2023
NorthSea Therapeutics receives FDA rare paediatric disease designation for SEFA-6179 for the treatment of intestinal failure-associated liver disease
17 October 2023 - Rare paediatric disease designation underscores critical need for novel therapies to address IFALD. ...
Posted by Michael Wonder on 07 Oct 2023
FDA approves Arcutis’ Zoryve (roflumilast) 0.3% cream for treatment of psoriasis in children ages 6 to 11
6 October 2023 - Expanded indication for Zoryve provides new, steroid free topical for children 6 to 11 with plaque ...
Posted 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 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 ...