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RSS FeedPosted by Michael Wonder on 18 Dec 2023
EMA recommends arpraziquantel for treatment of schistosomiasis in pre-school aged children
17 December 2023 - Astellas contributed to the development of a paediatric formulation to treat schistosomiasis as a member of ...
Posted by Michael Wonder on 09 Dec 2023
Medicines lobby says the PBS has not kept up with advances — or approvals — in medicines
8 December 2023 - Little Jack Roche was five weeks old when diagnosed with cystic fibrosis. The medication that could help ...
Posted by Michael Wonder on 08 Dec 2023
FDA approves expanded use of Cresemba (isavuconazonium sulphate) in children with invasive Aspergillosis and invasive Mucormycosis
8 December 2023 - Approval based on results of two paediatric studies in children aged 1 to 17. ...
Posted by Michael Wonder on 06 Dec 2023
Neurocrine Biosciences receives breakthrough therapy designation from US FDA for crinecerfont in congenital adrenal hyperplasia
5 December 2023 - Crinecerfont new drug application submission planned in 2024. ...
Posted by Michael Wonder on 04 Dec 2023
Vosoritide in achondroplasia: indication of an additional clinical benefit for children aged two and over
1 December 2023 - Two small RCTs with children of different ages together make it possible to make one statement: the ...
Posted by Michael Wonder on 02 Dec 2023
Health Canada grants market authorisation for Kalydeco (ivacaftor) for eligible children with cystic fibrosis ages 2 months and older
29 November 2023 - Vertex Pharmaceuticals today announced that Health Canada has granted market authorisation for the expanded use of ...
Posted by Michael Wonder on 30 Nov 2023
Burosumab for the treatment of adults with X-linked hypophosphataemia
29 November 2023 - The Department of Health and Social Care has asked NICE to produce guidance on the use ...
Posted by Michael Wonder on 28 Nov 2023
Sernova receives orphan drug and rare paediatric disease designations for its haemophilia A program from FDA
27 November 2023 - Sernova today announced the US FDA has granted both orphan drug designation and rare paediatric disease designation ...
Posted by Michael Wonder on 23 Nov 2023
Praxis Precision Medicines receives PRIME designation from the EMA for elsunersen (PRAX-222) for treatment of SCN2A gain of function developmental epilepsies
16 November 2023 - IRE) -- Praxis Precision Medicines today announced that the EMA has awarded its Priority Medicines (PRIME) designation ...
Posted by Michael Wonder on 21 Nov 2023
MHRA extends licence of Vertex’s cystic fibrosis drugs for young children
17 November 2023 - The MHRA has extended the licence of Vertex Pharmaceuticals’ cystic fibrosis drugs for children aged two ...
Posted by Michael Wonder on 09 Nov 2023
PHARMAC asked to reconsider funding RSV drug for at risk babies
9 November 2023 - Paediatricians and neonatal experts are pleading with PHARMAC to rethink its decision to stop funding a ...
Posted by Michael Wonder on 01 Nov 2023
X4 Pharmaceuticals announces FDA acceptance with priority review of US NDA for mavorixafor in WHIM syndrome
31 October 2023 - FDA sets a PDUFA target action date of 30 April 2024. ...
Posted 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 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 ...