Blog
RSS FeedPosted by Michael Wonder on 28 Aug 2019
Promoting paediatric drug research and labelling — outcomes of legislation
28 August 2019 - Congress has modified regulations of the FDA to promote research assessing the efficacy and safety of drugs ...
Posted by Michael Wonder on 20 Aug 2019
Teva launches generic version of EpiPen for young children
20 August 2019 - Teva Pharmaceutical Industries on Tuesday made its generic version of Mylan’s EpiPen for young children available in ...
Posted by Michael Wonder on 14 Aug 2019
This drug will save children’s lives. It costs $2 million.
13 August 2019 - Safety, innovation and affordability need not be mutually exclusive goals for cutting-edge treatments. ...
Posted by Michael Wonder on 05 Aug 2019
FDA issues draft guidance regarding clinical trial design for newborns
31 July 2019 - Today, the U.S. Food and Drug Administration issued the draft guidance, General Clinical Pharmacology Considerations for Neonatal ...
Posted by Michael Wonder on 01 Aug 2019
Ultragenyx announces submission of new drug application to FDA for UX007 (triheptanoin) for treatment of long-chain fatty acid oxidation disorders
1 August 2019 - Ultragenyx today announced that it has submitted to the U.S. FDA a new drug application for UX007 ...
Posted by Michael Wonder on 31 Jul 2019
Momenta Pharmaceuticals announces fast track designation for M281 (nipocalimab) in haemolytic disease of the foetus and newborn
30 July 2019 - Momenta Pharmaceuticals today announced that the U.S. FDA has granted fast track designation for M281, Momenta’s ...
Posted by Michael Wonder on 29 Jul 2019
FDA updates draft guidance on rare paediatric disease priority review voucher program
29 July 2019 - The U.S. FDA today issued a revised draft guidance, Rare Paediatric Disease Priority Review Vouchers – ...
Posted by Michael Wonder on 18 Jul 2019
LifeMax receives rare paediatric disease designation for LM-030 for the treatment of Netherton syndrome
18 July 2019 - LifeMax Laboratories today announced that the FDA granted rare paediatric disease designation to LM-030, an investigational therapy ...
Posted by Michael Wonder on 17 Jul 2019
LogicBio Therapeutics receives rare paediatric disease designation for LB-001 for the treatment of methylmalonic acidaemia
16 July 2019 - LogicBio Therapeutics today announced the U.S. FDA has granted rare paediatric disease designation to LB-001, a ...
Posted by Michael Wonder on 10 Jul 2019
FDA approves Katerzia, the first and only amlodipine oral suspension for paediatric patients 6 years of age and older
10 July 2019 - Azurity Pharmaceuticals announced today that the U.S. FDA has approved Katerzia (amlodipine) 1 mg/mL oral suspension, ...
Posted by Michael Wonder on 22 Jun 2019
FDA approves Botox (onabotulinumtoxinA) for paediatric patients with upper limb spasticity
21 June 2019 - Approval marks 10th Botox therapeutic indication in its 30th anniversary year. ...
Posted by Michael Wonder on 21 Jun 2019
FDA expands approval of treatment for cystic fibrosis to include patients ages 6 and older
21 June 2019 - The U.S. FDA today expanded the indication for Symdeko (a combination of tezacaftor/ivacaftor) tablets for treatment of ...
Posted by Michael Wonder on 17 Jun 2019
FDA approves new treatment for paediatric patients with type 2 diabetes
17 June 2019 - The U.S. Food and Drug Administration today approved Victoza (liraglutide) injection for treatment of paediatric patients 10 ...
Posted by Michael Wonder on 13 Jun 2019
FDA grants priority review to Genentech’s Rituxan (rituximab) in children with two rare blood vessel disorders
12 June 2019 - If approved, this would be the first paediatric indication for Rituxan. ...
Posted by Michael Wonder on 08 Jun 2019
PTC Therapeutics receives FDA approval for the expansion of the Emflaza (deflazacort) labeling to include patients 2-5 years of age
7 June 2019 - Emflaza is the only approved treatment for all patients two and over living with Duchenne muscular dystrophy. ...