Blog
RSS FeedPosted by Michael Wonder on 09 Oct 2020
U.S. FDA grants rare paediatric disease designation to Cerecin’s investigational drug tricaprilin for the treatment of infantile spasms
8 October 2020 - Cerecin announced that the U.S. FDA has granted rare paediatric disease designation to tricaprilin, an investigational drug ...
Posted by Michael Wonder on 08 Oct 2020
Association between FDA and EMA expedited approval programs and therapeutic value of new medicines: retrospective cohort study
7 October 2020 - The investigators of the study set out to characterise the therapeutic value of new drugs approved ...
Posted by Michael Wonder on 08 Oct 2020
Y-mAbs’ nivatrotamab for the treatment of patients with neuroblastoma granted orphan drug designation and rare paediatric disease designation by FDA
7 October 2020 - Y-mAbs Therapeutics today announced that the FDA has granted orphan drug designation and rare paediatric disease ...
Posted by Michael Wonder on 07 Oct 2020
Oncternal Therapeutics receives rare paediatric disease designation from U.S. FDA for TK216 for treatment of Ewing sarcoma
6 October 2020 - Oncternal Therapeutics today announced that the U.S. FDA has granted rare paediatric disease designation for TK216, an ...
Posted by Michael Wonder on 07 Oct 2020
Aegle Therapeutics receives rare paediatric disease designation from the FDA for AGLE-102 for patients with dystrophic epidermolysis bullosa
6 October 2020 - -- Aegle Therapeutics today announced that the FDA has granted rare paediatric disease designation to ...
Posted by Michael Wonder on 06 Oct 2020
FDA announces all time low rates for FY2021 priority review vouchers
5 October 2020 - The US FDA has announced historic low rates for the fiscal year 2021 fees to use ...
Posted by Michael Wonder on 06 Oct 2020
FDA grants GlycoMimetics rare paediatric disease designation for rivipansel for treatment of sickle cell disease
5 October 2020 - GlycoMimetics today announced that the U.S. FDA has granted the Company a rare paediatric disease designation for ...
Posted by Michael Wonder on 01 Oct 2020
US FDA grants rare paediatric disease designation to ATL1102 for the treatment of DMD
30 September 2020 - Priority review voucher may be utilised to expedite marketing authorisation review by FDA. ...
Posted by Michael Wonder on 29 Sep 2020
BridgeBio Pharma and affiliate Origin Biosciences announces FDA acceptance of its new drug application for fosdenopterin for the treatment of MoCD type A
29 September 2020 - Application accepted under priority review designation with breakthrough therapy designation and rare paediatric disease designation previously granted. ...
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 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 24 Sep 2020
FDA grants Spirovant Sciences orphan drug and rare paediatric disease designations for SPIRO-2101 for treatment of cystic fibrosis
24 September 2020 - Spirovant Sciences today announced that the U.S. FDA has granted orphan drug and rare paediatric disease ...
Posted by Michael Wonder on 23 Sep 2020
FDA grants RMAT designation to MultiStem cell therapy for the treatment of acute respiratory distress syndrome
23 September 2020 - ARDS program well positioned for an expedited path to commercialisation with RMAT and fast track designation. ...