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RSS FeedPosted by Michael Wonder on 28 Sep 2020
Khondrion receives rare paediatric disease designation for sonlicromanol from US FDA
28 September 2020 - Designation granted for the treatment of MELAS (mitochondrial encephalopathy, lactic acidosis, and stroke-like episodes) syndrome. ...
Posted by Michael Wonder on 26 Sep 2020
FDA grants rare paediatric disease designation to volasertib for rhabdomyosarcoma
25 September 2020 - Oncoheroes Biosciences is pleased to announce that the United States FDA has granted the designation of rare ...
Posted by Michael Wonder on 26 Sep 2020
FDA approves Kalydeco (ivacaftor) as first and only CFTR modulator to treat eligible infants with CF as early as four months of age
25 September 2020 - Approval provides opportunity to treat the underlying cause of cystic fibrosis earlier than ever before. ...
Posted by Michael Wonder on 25 Sep 2020
Innova Therapeutics receives rare paediatric disease designation from the FDA for IVT-8086 for the treatment of osteosarcoma
24 September 2020 - Innova Therapeutics Inc., a biopharmaceutical company committed to developing innovative cancer therapies for patients who have inadequate ...
Posted by Michael Wonder on 25 Sep 2020
Mereo BioPharma receives FDA rare paediatric disease designation for setrusumab for the treatment of osteogenesis imperfecta
24 September 2020 - Mereo BioPharma today announces that the U.S. FDA has granted rare paediatric disease designation to setrusumab for ...
Posted by Michael Wonder on 24 Sep 2020
FDA grants AT-007 paediatric rare disease designation and orphan designation for treatment of PMM2-CDG
24 September 2020 - Applied Therapeutics announced today that the U.S. FDA has granted AT-007 both paediatric rare disease designation and ...
Posted by Michael Wonder on 23 Sep 2020
FDA accepts supplemental new drug application for Pfizer’s Xalkori (crizotinib) for the treatment of paediatric ALK positive anaplastic large cell lymphoma
23 September 2020 - If approved, Xalkori would be the first biomarker driven therapy for paediatric ALK positive anaplastic large cell ...
Posted by Michael Wonder on 21 Sep 2020
Crinetics Pharmaceuticals receives rare paediatric disease designation from FDA for CRN04777 for the treatment of congenital hyperinsulinism
21 September 2020 - Crinetics Pharmaceuticals today announced that the U.S. Food and Drug FDA has granted rare paediatric disease designation ...
Posted by Michael Wonder on 16 Sep 2020
FDA granted paediatric disease designation for OXi-4503
16 September 2020 - Treatment of acute myeloid leukaemia due to genetic mutations that disproportionately affect paediatric patients. ...
Posted by Michael Wonder on 15 Sep 2020
Ziopharm Oncology granted rare paediatric disease designation for controlled IL-12 for the treatment of DIPG
14 September 2020 - -- Ziopharm Oncology today announced that the U.S. FDA has granted rare paediatric disease designation to ...
Posted by Michael Wonder on 09 Sep 2020
Clarity Pharmaceuticals announces the US FDA grants rare paediatric disease designation to 64 Cu sartate, a diagnostic for the clinical management of neuroblastoma
9 September 2020 - Clarity Pharmaceuticals is pleased to announce that the U.S. FDA has granted rare paediatric disease designation to ...
Posted by Michael Wonder on 27 Aug 2020
Editas Medicine receives rare paediatric disease designation for EDIT-301 for the treatment of sickle cell disease
24 August 2020 - Editas Medicine today announced that the U.S. FDA has granted rare paediatric disease designation for EDIT-301, an ...
Posted by Michael Wonder on 21 Aug 2020
BioMarin submits new drug application to U.S. FDA for vosoritide to treat children with achondroplasia
20 August 2020 - Potential first treatment for achondroplasia in the United States. ...
Posted by Michael Wonder on 20 Aug 2020
FDA approves first paediatric indication for Xeomin (incobotulinumtoxinA) for the treatment of upper limb spasticity, excluding spasticity caused by cerebral palsy
19 August 2020 - Xeomin was also granted FDA priority review for an application for paediatric sialorrhea, reinforcing Merz Therapeutics’ commitment ...
Posted by Michael Wonder on 20 Aug 2020
U.S. FDA grants rare paediatric disease designation to AmideBio’s glucagon analog for the treatment of congenital hyperinsulinism
20 August 2020 - AmideBio announced today that the US FDA Offices of Pediatric Therapeutics and Orphan Products Development granted a ...