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RSS FeedPosted 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 ...
Posted by Michael Wonder on 06 Oct 2022
US FDA accepts Takeda’s supplemental biologics license application for use of Takzyro (lanadelumab-flyo) to prevent hereditary angioedema attacks in children 2 years of age and older
5 October 2022 - Filing is based on data from Phase 3 SPRING study in paediatric patients 2 to <12 years ...
Posted by Michael Wonder on 29 Sep 2022
Catalyst Pharmaceuticals announces FDA approval of supplemental new drug application for Firdapse expanding patient population to include paediatric patients
29 September 2022 - Firdapse is now a treatment option for all LEMS patients 6 years of age and older in ...
Posted by Michael Wonder on 24 Sep 2022
Moderna asks FDA to authorise omicron Covid boosters for children as young as 6 years old
23 September 2022 - The Centers for Disease Control and Prevention, in a document published Tuesday, said it expects children to ...
Posted by Michael Wonder on 24 Sep 2022
FDA grants rare paediatric disease designation to dovitinib for osteosarcoma
22 September 2022 - Rare paediatric disease designation qualifies Oncoheroes to receive fast track review, and a priority review voucher at ...
Posted by Michael Wonder on 20 Sep 2022
FDA approves sodium thiosulfate to reduce the risk of ototoxicity associated with cisplatin in paediatric patients with localised, non-metastatic solid tumours
20 September 2022 - Today, the FDA approved sodium thiosulphate (Pedmark, Fennec Pharmaceuticals) to reduce the risk of ototoxicity associated ...