Blog
RSS FeedPosted by Michael Wonder on 14 Aug 2023
In the case of a devastating disease, the FDA weighs an experimental drug’s muddled data and a desperate need
14 August 2023 - Emma Albee knows the experimental drug she takes is not a cure. It won’t allow her ...
Posted by Michael Wonder on 10 Aug 2023
US FDA accepts Astellas' sNDA for Cresemba (isavuconazonium sulphate) in children
10 August 2023 - Astellas announced that the US FDA has accepted the company's supplemental new drug application for Cresemba (isavuconazonium ...
Posted by Michael Wonder on 07 Aug 2023
HuidaGene Therapeutics receives FDA rare paediatric disease designation for HG004 to treat inherited blindness
7 August 2023 - HG004 is a one time, direct to RPE treatment of inherited retinal disease caused by mutations in ...
Posted by Michael Wonder on 04 Aug 2023
Mesoblast receives complete response from US FDA for biologics license application for steroid-refractory acute graft versus host disease in children
4 August 2023 - Mesoblast Limited today announced that the US FDA has provided a complete response to its biologics license ...
Posted by Michael Wonder on 03 Aug 2023
Aardvark Therapeutics announces receipt of FDA rare paediatric disease designation for Prader-Willi syndrome
3 August 2023 - Aardvark's Phase 2 trial of oral ARD-101 in young adults with Prader-Willi syndrome is now open ...
Posted by Michael Wonder on 03 Aug 2023
US FDA approves Merck’s Ervebo (Ebola Zaire vaccine, live) for use in children 12 months of age and older
3 August 2023 - Merck continues progress in helping to protect people at risk of Zaire ebolavirus disease. ...
Posted by Michael Wonder on 28 Jul 2023
Janssen submits supplemental new drug application to US FDA seeking expanded paediatric indication for HIV-1 therapy Edurant
28 July 2023 - Parallel application also submitted to European Medicines Agency. ...
Posted by Michael Wonder on 25 Jul 2023
US FDA accepts supplemental new drug application for Ofev (nintedanib) for children and adolescents aged 6-17 years old with fibrosing interstitial lung disease
25 July 2023 - The supplemental new drug application is based on results from the InPedILD Phase III trial, which assessed ...
Posted by Michael Wonder on 20 Jul 2023
Advent Therapeutics receives FDA rare paediatric disease designation for retinol palmitate for prevention of bronchopulmonary dysplasia in premature infants
20 July 2023 - Advent plans FDA PDUFA meeting late 2023 to establish pathway for US approval of lead injectable ...
Posted by Michael Wonder on 17 Jul 2023
FDA approves new drug to prevent RSV in babies and toddlers
17 July 2023 - Today, the US FDA approved Beyfortus (nirsevimab-alip) for the prevention of respiratory syncytial virus lower respiratory tract ...
Posted by Michael Wonder on 10 Jul 2023
MyoPax receives rare paediatric disease designation from FDA for breakthrough regenerative cell product in exstrophy-epispadias complex
10 July 2023 - The US FDA granted a rare paediatric Disease designation to MyoPax.. ...
Posted by Michael Wonder on 07 Jul 2023
FDA grants rare paediatric disease designation to NS-089/NCNP-02 for the treatment of Duchenne muscular dystrophy
7 July 2023 - NS Pharma announced today the US FDA has granted rare paediatric disease designation to NS-089/NCNP-02 (brogidirsen) an ...
Posted by Michael Wonder on 06 Jul 2023
IPS Heart receives US FDA rare paediatric drug designation for ISX9-CPC stem cell therapy for treatment of cardiomyopathy associated with Danon disease
6 July 2023 - Marks third rare paediatric drug designation granted by FDA for pipeline candidate. ...
Posted by Michael Wonder on 28 Jun 2023
FDA approves Pfizer’s Ngenla, a long-acting once weekly treatment for paediatric growth hormone deficiency
28 June 2023 - New, longer-acting treatment offers option to reduce the frequency of injections for children with growth hormone ...
Posted by Michael Wonder on 22 Jun 2023
FDA approves first gene therapy for treatment of certain patients with Duchenne muscular dystrophy
22 June 2023 - Today, the US FDA approved Elevidys, the first gene therapy for the treatment of pediatric patients 4 ...