Blog
RSS FeedPosted by Michael Wonder on 04 Mar 2021
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 ...
Posted by Michael Wonder on 28 Feb 2021
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. ...
Posted by Michael Wonder on 26 Feb 2021
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. ...
Posted by Michael Wonder on 25 Feb 2021
NICE draft guidance recommends sapropterin for children with rare inherited metabolic condition phenylketonuria
25 February 2021 - NICE has today issued draft guidance for public consultation which recommends sapropterin (Kuvan, BioMarin) for treating phenylketonuria ...
Posted by Michael Wonder on 25 Feb 2021
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 ...
Posted by Michael Wonder on 24 Feb 2021
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. ...
Posted by Michael Wonder on 16 Feb 2021
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 ...
Posted by Michael Wonder on 15 Feb 2021
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 ...
Posted by Michael Wonder on 15 Feb 2021
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 ...
Posted by Michael Wonder on 12 Feb 2021
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 ...
Posted by Michael Wonder on 10 Feb 2021
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. ...
Posted by Michael Wonder on 09 Feb 2021
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 ...
Posted by Michael Wonder on 09 Feb 2021
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. ...
Posted by Michael Wonder on 05 Feb 2021
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 ...
Posted by Michael Wonder on 03 Feb 2021
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 ...