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 ...

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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. ...

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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 ...

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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 ...

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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 ...

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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 ...

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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 ...

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BioMarin receives positive CHMP opinion in Europe to expand use of Voxzogo (vosoritide) to treat children aged 4 months and older with achondroplasia

15 September 2023 - BioMarin today announced that the EMA's CHMP has adopted a positive opinion recommending marketing authorisation to expand ...

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Vertex receives CHMP positive opinion for Kaftrio (ivacaftor/tezacaftor/elexacaftor) in combination with ivacaftor for children with cystic fibrosis ages 2 through 5

15 September 2023 - If approved, more than 1,200 children would be newly eligible for a medicine that could treat ...

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European Commission expands Merck’s Ervebo [Ebola Zaire vaccine, live] indication to include children 1 year of age and older

7 September 2023 - Milestone signifies on-going effort to help prepare for outbreaks of Zaire ebolavirus. ...

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Mum vows to keep pushing for fenfluramine PBS listing for Dravet syndrome, a severe form of epilepsy

5 September 2023 - Families calling for greater access to a medication that helps manage a severe form of epilepsy ...

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European Commission approves Roche’s Evrysdi for babies under two months old with spinal muscular atrophy

29 August 2023 - Approval is based on interim data from ongoing RAINBOWFISH trial showing majority of Evrysdi-treated babies were able ...

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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 ...

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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. ...

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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 ...

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