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RSS FeedPosted by Michael Wonder on 20 Nov 2019
Newron receives FDA rare paediatric disease designation for sarizotan for the treatment of Rett syndrome
19 November 2019 - Newron announced today that the U.S. FDA has granted the rare paediatric disease designation for sarizotan, ...
Posted by Michael Wonder on 14 Nov 2019
Anavex Life Sciences receives rare paediatric disease designation from FDA for Anavex 2-73 (blarcamesine) for the treatment of Rett syndrome
14 November 2019 - Anavex Life Sciences today announced that it received the rare paediatric disease designation from the U.S. FDA ...
Posted by Michael Wonder on 24 Oct 2019
FDA approves Botox (onabotulinumtoxinA) for paediatric patients with lower limb spasticity, excluding spasticity caused by cerebral palsy
24 October 2019 - Approval marks 11th Botox therapeutic indication and comes 30 years after the first indications were approved. ...
Posted by Michael Wonder on 16 Oct 2019
ProQR receives rare paediatric disease designation from FDA for sepofarsen for the treatment of LCA10
15 October 2019 - ProQR Therapeutics today announced that it received rare paediatric disease designation from the U.S. FDA for ...
Posted by Michael Wonder on 15 Oct 2019
Faced with a drug shortfall, doctors scramble to treat children with cancer
14 October 2019 - A critical chemotherapy medication is in short supply, and physicians say there is no appropriate substitute. ...
Posted by Michael Wonder on 07 Oct 2019
Janssen submits application to U.S. FDA seeking approval of Stelera (ustekinumab) for the treatment of paediatric patients with moderate to severe plaque psoriasis
7 October 2019 - The Janssen Pharmaceutical Companies of Johnson & Johnson today announced the submission of a supplemental biologics license ...
Posted by Michael Wonder on 03 Oct 2019
Novartis Entresto receives FDA approval for paediatric heart failure, helping to address critical unmet need for treatment options
1 October 2019 - Entresto is now approved for the treatment of paediatric patients aged 1 year and older with symptomatic ...
Posted by Michael Wonder on 27 Sep 2019
FDA approves first treatment for children with rare diseases that cause inflammation of small blood vessels
27 September 2019 - The U.S. FDA today approved Rituxan (rituximab) injection to treat granulomatosis with polyangiitis (GPA) and microscopic polyangiitis ...
Posted by Michael Wonder on 12 Sep 2019
Nucala is the first biologic approved in the US for six to 11-year-old children with severe eosinophilic asthma
12 September 2019 - GlaxoSmithKline today announced that the US FDA has approved Nucala (mepolizumab) for use in children as young ...
Posted by Michael Wonder on 04 Sep 2019
GeneTx and Ultragenyx announce orphan drug designation and rare paediatric disease designation for GTX-102
3 September 2019 - GeneTx Biotherapeutics and Ultragenyx today announced that the U.S. FDA has granted orphan drug designation and rare ...
Posted 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 ...