Precision BioSciences receives FDA rare paediatric disease designation for PBGENE-DMD for the treatment of Duchenne muscular dystrophy

25 June 2025 - Precision BioSciences today announced that the US FDA has granted rare paediatric disease designation for PBGENE-DMD for ...

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Quoin Pharmaceuticals announces FDA grants rare paediatric disease designation for QRX003 in Netherton syndrome

24 June 2025 - Quoin Pharmaceuticals today announced that the US FDA has granted rare paediatric disease designation for the ...

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FDA approves Benlysta (belimumab) auto-injector for children with active lupus nephritis

24 June 2025 - With this approval, paediatric patients aged five years and older with active lupus nephritis will have ...

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Johnson & Johnson seeks US FDA approval of Stelara (ustekinumab) for the treatment of paediatric Crohn’s disease

17 June 2025 - Supported by 52 week data from the Phase 3 UNITI-Jr study, submission aims to expand Stelara indication ...

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Celltrion announces US FDA approval of additional presentation of Steqeyma (ustekinumab-stba), expanding dosing options for pediatric patients

15 June 2025 - Approval of 45 mg/0.5 mL solution in a single-dose vial for subcutaneous injection expands dosing flexibility for ...

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Sydnexis announces European Commission approval of SYD-101, the first and only pharmaceutical treatment for slowing the progression of paediatric myopia

5 June 2025 - Exclusive licensing partner Santen will commercialise SYD-101 under the brand name Ryjunea in the EU. ...

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RSV prophylaxis with nirsevimab: benefits for healthy children in the first RSV season

2 June 2025 - Children immunised with the antibody nirsevimab are significantly less likely to develop RSV-related lower respiratory tract infections. ...

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FDA accepts TransCon CNP NDA for priority review

2 June 2025 - Ascendis Pharma today announced that the US FDA has accepted for priority review its new drug application ...

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Ocugen announces rare paediatric disease designation granted for OCU410ST—modifier gene therapy for the treatment of Stargardt disease

27 May 2025 - Ocugen today announced that the US FDA has granted rare paediatric disease designation for OCU410ST for the ...

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Relief Therapeutics receives FDA rare paediatric disease designation for RLF-TD011

22 May 2025 - Relief Therapeutics today announced that the US FDA has granted rare paediatric disease designation to RLF-TD011 ...

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US FDA grants approval for Jivi anti-hemophilic factor (recombinant), PEGylated-aucl in paediatric patients 7 to under 12 years of age with haemophilia A

19 May 2025 - Bayer announced that the US FDA has approved Jivi, a recombinant DNA-derived, extended half-life factor VIII ...

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Kaerus Bioscience's lead candidate KER-0193 granted orphan drug designation and rare paediatric drug designation by US FDA for treatment of Fragile X syndrome

14 May 2025 - Kaerus Bioscience today announces that its lead candidate KER-0193 has been granted both orphan drug designation and ...

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FDA grants regenerative medicine advanced therapy designation for BrainChild Bio’s B7-H3 CAR T-cell therapy for incurable paediatric brain tumours

15 May 2025 - BrainChild Bio advancing BCB-276 towards BLA submission with a single pivotal Phase 2 trial planned to ...

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BioCryst announces FDA acceptance of NDA for Orladeyo (berotralstat) oral granules in patients with hereditary angioedema aged 2 to 11 years

14 May 2025 - FDA grants priority review of application, with PDUFA target action date of 12 September 2025. ...

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Itulatek approved for treatment of children and adolescents in Canada

8 May 2025 - ALK today announced that Health Canada has approved ALK’s regulatory filing for Itulatek (tree pollen sublingual allergy ...

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