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RSS FeedPosted 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 ...
Posted by Michael Wonder on 20 Sep 2022
AvroBio receives rare paediatric disease designation from US FDA for first gene therapy in development for cystinosis
20 September 2022 - AVR-RD-04 has previously received orphan drug designation from FDA and EMA. ...
Posted by Michael Wonder on 19 Sep 2022
FDA grants Radiopharm Theranostics DUNP19 product rare paediatric disease designation
16 September 2022 - Radiopharm Theranostics has been granted rare paediatric disease designation by the US FDA for its DUNP19 ...
Posted by Michael Wonder on 15 Sep 2022
Aro Biotherapeutics receives FDA rare paediatric drug designation for ABX1100 for the treatment of Pompe disease
15 September 2022 - Aro Biotherapeutics today announced that the US FDA granted rare paediatric drug designation for ABX1100, an investigational ...
Posted by Michael Wonder on 02 Sep 2022
Vertex announces U.S. FDA approval for Orkambi (lumacaftor/ivacaftor) in children with cystic fibrosis ages 12 to <24 months
2 September 2022 - With this approval, approximately 300 children with two copies of the F508del mutation will have a ...
Posted by Michael Wonder on 31 Aug 2022
US FDA grants fast track designation for Longeveron’s Lomecel-B product for treatment of hypoplastic left heart syndrome in infants
31 August 2022 - New designation may expedite FDA review and potential approval to address this life-threatening heart condition affecting ...
Posted by Michael Wonder on 25 Aug 2022
Children with chronic graft versus host disease may now be prescribed Imbruvica (ibrutinib)
24 August 2022 - This is the first approved treatment option for children with cGVHD under 12 years of age and ...
Posted by Michael Wonder on 24 Aug 2022
FDA approves ibrutinib for paediatric patients with chronic graft versus host disease, including a new oral suspension
24 August 2022 - Today, the FDA approved ibrutinib (Imbruvica, Pharmacyclics) for paediatric patients ≥ 1 year of age with ...
Posted by Michael Wonder on 11 Aug 2022
Genentech announces FDA approval of Xofluza to treat influenza in children aged five and older
11 August 2022 - The FDA also approved Xofluza to prevent influenza in children aged five and older following contact with ...
Posted by Michael Wonder on 01 Aug 2022
Stelara (ustekinumab) approved by the U.S. FDA to treat paediatric patients with active psoriatic arthritis
1 August 2022 - As the first and only biologic targeting both cytokines interleukin-12 and interleukin-23, Stelara provides a new therapeutic ...
Posted by Michael Wonder on 27 Jul 2022
GSK announces US FDA approval of Benlysta (belimumab) for paediatric patients with active lupus nephritis
27 July 2022 - Benlysta is now the first and only biologic approved for adults and children who have lupus ...