Blog
RSS FeedPosted 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 ...
Posted by Michael Wonder on 20 Jul 2022
FDA approves Qsymia for the treatment of obesity in adolescents ages 12-17
20 July 2022 - Vivus today announced that the U.S. FDA approved Qsymia (phentermine and topiramate extended-release capsules) for use in ...
Posted by Michael Wonder on 19 Jul 2022
Wugen receives US FDA fast track and rare paediatric disease designations for WU-CART-007 for the treatment of R/R T-ALL/LBL
19 July 2022 - Wugen, today announced that the U.S. FDA has granted fast track designation and rare paediatric disease ...
Posted by Michael Wonder on 09 Jul 2022
Pfizer and BioNTech announce U.S. FDA approval of their COVID-19 vaccine Comirnaty for adolescents 12 through 15 years of age
8 July 2022 - Approval of the two dose primary series is based on the totality of data through six months ...
Posted by Michael Wonder on 07 Jul 2022
US FDA awards rare paediatric disease designation to paxalisib for AT/RT, a rare form of childhood brain cancer
6 July 2022 - Kazia Therapeutics is pleased to announce that the U.S. FDA has awarded rare paediatric disease designation ...
Posted by Michael Wonder on 27 Jun 2022
FDA approves treatment for chronic weight management in certain paediatric patients aged 12 years and older
27 June 2022 - The U.S. FD has approved a supplemental indication for Qsymia (phentermine and topiramate extended-release capsules) for chronic ...