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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 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 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 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 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 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 21 Oct 2023
Rezolute receives Priority Medicines (PRIME) eligibility from EMA for enhanced regulatory support of RZ358 in congenital hyperinsulinism
17 October 2023 - PRIME eligibility granted based on key positive data from the Phase 2B (RIZE) study and current ...
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 18 Oct 2023
Health Canada grants market authorisation for Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor) in children with cystic fibrosis ages 2 to 5 years with at least one F508del mutation
17 October 2023 - Approximately 330 children with cystic fibrosis ages 2-5 years are now eligible for a medicine that ...
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 ...