FDA accepts Dupixent (dupilumab) for review in children with moderate to severe asthma

4 March 2021 - Submission supported by data demonstrating Dupixent significantly reduced severe asthma attacks and is the only biologic to ...

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EMA accepts marketing application for somatrogon to treat paediatric patients with growth hormone deficiency

26 February 2021 - If approved, somatrogon will serve as a once-weekly treatment option. ...

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Retrotope granted rare paediatric disease designation from FDA for lead development candidate, RT001, in two life-threatening neurodegenerative indications

25 February 2021 - RT001 also granted fast track designation by FDA in Friedreich’s ataxia. ...

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NICE draft guidance recommends sapropterin for children with rare inherited metabolic condition phenylketonuria

25 February 2021 - NICE has today issued draft guidance for public consultation which recommends sapropterin (Kuvan, BioMarin) for treating phenylketonuria ...

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Humira (adalimumab) receives FDA approval to treat paediatric patients living with moderately to severely active ulcerative colitis

24 February 2021 - Humira (adalimumab) is the first and only subcutaneous biologic treatment option for paediatric patients from 5 years ...

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Health Canada approves Dupixent (dupilumab) as the first biologic for the treatment of moderate to severe atopic dermatitis in children aged 6 to 11 years

23 February 2021 - Dupixent is the only biologic medicine approved to treat atopic dermatitis for this patient population. ...

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Forge Biologics receives FDA fast track, orphan drug, and rare paediatric disease designations for FBX-101 gene therapy for patients with Krabbe disease

16 February 2021 - FBX-101 is a first-in-human gene therapy utilising an adeno-associated virus to deliver a functioning copy of the ...

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Polaryx Therapeutics receives both rare paediatric disease and orphan drug designations for the treatment of Krabbe disease with PLX-300

10 February 2021 - Polaryx Therapeutics announced today that it has received both rare paediatric disease and orphan drug designations for ...

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It’s time to fast track innovation in medical devices for children

11 February 2021 - When a baby arrived at Children’s National Hospital with a critically underdeveloped heart, it was clear ...

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Rescindo Therapeutics’ RSC-57 receives FDA orphan drug designation and rare paediatric disease designation for Kabuki syndrome

12 February 2021 - Rescindo Therapeutics announced today that the U.S. FDA has granted orphan drug designation and rare paediatric disease ...

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Botox (onabotulinumtoxinA) receives FDA approval for paediatric detrusor overactivity associated with a neurologic condition

10 February 2021 - Milestone marks 12th U.S. therapeutic indication for Botox. ...

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Supernus resubmits new drug application for SPN-812 for the treatment of ADHD in paediatric patients

8 February 2021 - Supernus Pharmaceuticals today announced it has resubmitted its ew drug application for SPN-812 for the treatment of ...

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Teen thriving on PHARMAC funded medicine

9 February 2021 - 14 year old Bruno Cettina is living his best life thanks to a medicine funded by PHARMAC.  ...

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FDA grants Actinogen rare paediatric disease designation in Fragile X syndrome

5 February 2021 - Actinogen is pleased to announce that the United States FDA has granted Actinogen’s drug Xanamem rare paediatric ...

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BioCryst announces FDA approval of supplemental new drug application for Rapivab expanding patient population to include children six months and older

3 February 2021 - BioCryst Pharmaceuticals today announced that the U.S. FDA has approved a supplemental new drug application for ...

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