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RSS FeedPosted by Michael Wonder on 08 Mar 2023
FDA accepts Mesoblast’s resubmission of the biologic license application for remestemcel-L in children with steroid-refractory acute graft versus host disease as a complete response
7 March 2023 - If approved, remestemcel-L will be the first allogeneic “off the shelf” cellular medicine in the US and ...
Posted by Michael Wonder on 08 Mar 2023
FDA accepts BioMarin's supplemental new drug application to expand use of Voxzogo (vosoritide) for injection to treat children with achondroplasia under the age of 5
7 March 2023 - FDA set PDUFA target action date of 21 October 2023. ...
Posted by Michael Wonder on 01 Mar 2023
Pfizer and BioNTech submit for US emergency use authorisation of Omicron BA.4/BA.5 adapted bivalent COVID-19 booster in children under 5 years
1 March 2023 - Pfizer and BioNTech today submitted an application to the US FDA for emergency use authorisation of ...
Posted by Michael Wonder on 13 Feb 2023
Ironwood Pharmaceuticals announces FDA filing acceptance and priority review of supplemental new drug application for Linzess (linaclotide) for functional constipation in children and adolescents ages 6-17 years old
13 February 2023 - FDA assigns second quarter 2023 target action date. ...
Posted by Michael Wonder on 10 Feb 2023
FDA approves Pfizer's supplemental new drug application for Cibinqo (abrocitinib)
10 February 2023 - Label expansion for Cibinqo provides new systemic oral option for adolescents (12 to <18 years) with ...
Posted by Michael Wonder on 09 Feb 2023
Eylea (aflibercept) injection approved as the first pharmacological treatment for preterm infants with retinopathy of prematurity by the FDA
8 February 2023 - Eylea now approved to treat five retinal conditions caused by ocular angiogenesis. ...
Posted by Michael Wonder on 04 Feb 2023
US FDA approves Takeda’s Takhyzro (lanadelumab-flyo) to prevent hereditary angioedema attacks in children 2 years of age and older
3 February 2023 - Approval supported by extrapolation of efficacy data from the Phase 3 HELP study with additional data from ...
Posted by Michael Wonder on 01 Feb 2023
FLAG Therapeutics receives rare paediatric disease designation for FLAG-003 for the treatment of children with diffuse intrinsic pontine glioma
31 January 2023 - FLAG Therapeutics announced today that FLAG-003, an investigational small molecule therapy for the treatment of diffuse intrinsic ...
Posted by Michael Wonder on 01 Feb 2023
Mesoblast resubmits biologic license application to FDA for remestemcel-L in children with steroid-refractory acute graft versus host disease
31 January 2023 - New 4 year data from the Phase 3 trial shows durable long term survival outcomes. ...
Posted by Michael Wonder on 25 Jan 2023
US FDA approves ALK's Odactra (house dust mite allergen extract) tablet for sublingual use as immunotherapy for adolescents
25 January 2023 - Odactra is now indicated to treat house dust mite-induced allergic rhinitis, with or without conjunctivitis, in persons ...
Posted by Michael Wonder on 19 Jan 2023
Synlogic receives rare paediatric disease designation from FDA for SYNB1934 for phenylketonuria
19 January 2023 - Synlogic today announced that SYNB1934 was granted rare paediatric disease designation by the US FDA for the ...
Posted by Michael Wonder on 19 Jan 2023
FDA grants rare paediatric disease status to GC Pharma’s San Filippo syndrome therapy
18 January 2023 - GC Pharma said on Wednesday that its San Filippo Syndrome type A treatment, jointly being developed ...
Posted by Michael Wonder on 17 Jan 2023
Orphagen Pharmaceuticals receives rare paediatric disease designation from FDA for OR-449 for the treatment of paediatric adrenocortical carcinoma
17 January 2023 - Orphagen Pharmaceuticals today announced that the US FDA has granted rare paediatric disease designation for OR-449 ...
Posted by Michael Wonder on 06 Jan 2023
AUM Biosciences receives US FDA rare paediatric disease designation for AUM302 for treatment of neuroblastoma
5 January 2023 - AUM Biosciences announced today that the US FDA has granted a rare paediatric disease designation for its ...
Posted by Michael Wonder on 05 Jan 2023
Nirsevimab US regulatory submission accepted for the prevention of RSV lower respiratory tract disease in infants and children up to age 24 months
5 January 2023 - Submission supported by comprehensive clinical trial programme, which demonstrated protection against RSV disease through the RSV ...