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RSS FeedPosted by Michael Wonder on 17 Jan 2024
Atsena Therapeutics receives rare paediatric disease designation from FDA for ATSN-101 gene therapy for GUCY2D associated Leber congenital amaurosis
16 January 2023 - Atsena Therapeutics today announced the US FDA has granted rare paediatric disease designation to ATSN-101, the company’s ...
Posted by Michael Wonder on 14 Jan 2024
Drug Farm receives rare paediatric disease designation from the US FDA for DF-003 to treat ROSAH syndrome
13 January 2024 - Drug Farm announced that the US FDA has granted rare paediatric disease designation for the company’s ...
Posted by Michael Wonder on 18 Dec 2023
EndeavorRx, world’s first and only prescription video game treatment, secures FDA label expansion for paediatric ADHD patients aged 13-17
18 December 2023 - Akili’s clinically proven digital medicine now authorised for children 8-17, opening option to more patients as ...
Posted by Michael Wonder on 18 Dec 2023
Leo Pharma announces US FDA approval of Adbry (tralokinumab-ldrm) for the treatment of moderate to severe atopic dermatitis in paediatric patients aged 12-17 years
15 December 2023 - In the ECZTRA 6 trial, significantly more paediatric patients met the primary and key secondary endpoints 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 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 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 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 ...