Blog
RSS FeedPosted by Michael Wonder on 04 Jan 2023
European Medicines Agency validates application for extension of indication for Vozogo (vosoritide) for injection to treat children with achondroplasia under the age of 2
4 January 2023 - BioMarin submits supplemental new drug application to US FDA to expand label to treat children with achondroplasia ...
Posted by Michael Wonder on 30 Dec 2022
FDA approves once weekly Wegovy injection for the treatment of obesity in teens aged 12 years and older
24 December 2022 - New indication based on clinical trial that demonstrated a 16.1% reduction in body mass index (BMI) in ...
Posted by Michael Wonder on 17 Dec 2022
AbbVie submits supplemental new drug application to US FDA to support new indication of Linzess (linaclotide) for functional constipation in children and adolescents 6 to 17 years of age
16 December 2022 - Submission is based on positive Phase 3 study data demonstrating linaclotide (72 mcg) resulted in increases ...
Posted by Michael Wonder on 08 Dec 2022
FDA authorises updated (bivalent) COVID-19 vaccines for children down to 6 months of age
8 December 2022 - Today, the US FDA amended the emergency use authorisations of the updated (bivalent) Moderna and Pfizer-BioNTech COVID-19 ...
Posted by Michael Wonder on 06 Dec 2022
Pfizer and BioNTech submit application to US FDA for emergency use authorisation of Omicron BA.4/BA.5 adapted bivalent COVID-19 vaccine in children under 5 years
5 December 2022 - Pfizer andBioNTech today announced that the companies have submitted an application to the US FDA for ...
Posted by Michael Wonder on 15 Nov 2022
PerkinElmer announces its EONIS SCID-SMA kit is first to receive marketing authorisation by US FDA for SMA screening in newborns
14 November 2022 - Building on its contributions to newborn screening globally, PerkinElmer’s latest FDA authorised assay enables the simultaneous ...
Posted by Michael Wonder on 11 Nov 2022
Akari Therapeutics granted FDA rare paediatric disease designation of nomacopan for the treatment of paediatric HSCT-TMA
10 November 2022 - Akari Therapeutics today announced the US FDA has granted the rare paediatric disease designation to nomacopan for ...
Posted by Michael Wonder on 10 Nov 2022
Seagen announces US FDA approval of new indication for Adcetris (brentuximab vedotin) for children with previously untreated high risk Hodgkin's lymphoma
10 November 2022 - Approval based on Phase 3 clinical trial that demonstrated a 59% reduction in risk of disease ...
Posted by Michael Wonder on 07 Nov 2022
Sensorion announces US FDA grants rare paediatric disease designation to OTOF-GT for the treatment of otoferlin gene-mediated hearing loss
7 November 2022 - OTOF-GT targets the restoration of hearing in people living with otoferlin deficiency. ...
Posted by Michael Wonder on 02 Nov 2022
US FDA approves Vemlidy (tenofovir alafenamide) for treatment of chronic hepatitis B virus infection in paediatric patients
2 November 2022 - Approval expands on previous FDA approval of Vemlidy in adults living with this chronic liver disease. ...
Posted by Michael Wonder on 27 Oct 2022
AvroBio receives rare paediatric disease designation from the US FDA for first in class gene therapy for Gaucher disease
27 October 2022 - AVR-RD-02 has previously received fast track status from FDA, orphan drug designation in the US and EU, ...
Posted by Michael Wonder on 12 Oct 2022
Eylea (aflibercept) injection sBLA for treatment of retinopathy of prematurity accepted for FDA priority review
12 October 2022 - Regeneron Pharmaceuticals today announced the US FDA has accepted for priority review the supplemental biologics license application ...
Posted by Michael Wonder on 12 Oct 2022
FDA authorises Moderna and Pfizer-BioNTech bivalent COVID-19 vaccines for use as a booster dose in younger age groups
12 October 2022 - Today, the US FDA amended the emergency use authorisations of the Moderna COVID-19 Vaccine, Bivalent and the ...
Posted by Michael Wonder on 11 Oct 2022
Eligo Bioscience receives FDA orphan drug designation and rare paediatric disease designation for EB003 for the prevention of haemolytic uraemic syndrome with first in class CRISPR based medicine
11 October 2022 - Eligo Bioscience today announced the US FDA has granted orphan drug designation and rare paediatric disease designation ...
Posted by Michael Wonder on 07 Oct 2022
The critical role of academic clinical trials in paediatric cancer drug approvals: design, conduct, and fit for purpose data for positive regulatory decisions
6 October 2022 - For decades, academic clinical trials consortia have collaborated to optimise outcomes for childhood cancers through evaluating incremental ...