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RSS FeedPosted by Michael Wonder on 19 Oct 2020
Versantis receives FDA rare paediatric disease designation for VS-01 for the treatment of urea cycle disorders
19 October 2020 - Versantis today announced that the U.S. FDA has granted a rare paediatric disease designation to its lead ...
Posted by Michael Wonder on 15 Oct 2020
Taysha Gene Therapies receives rare paediatric disease designation and orphan drug designation for TSHA-102 as a treatment for Rett syndrome
14 October 2020 - Program leverages novel miRARE platform technology used to control transgene expression on a cellular basis. ...
Posted by Michael Wonder on 13 Oct 2020
Oxular receives rare paediatric disease and orphan drug designations for retinoblastoma treatment
12 October 2020 - Provides furthered momentum for OXU-003 development programme. ...
Posted by Michael Wonder on 11 Oct 2020
Axovant Gene Therapies receives rare paediatric disease designation for AXO-AAV-GM1 for GM1 gangliosidosis
9 October 2020 - Company on-track to report data from on-going Phase 1/2 clinical study in Q4 2020. ...
Posted by Michael Wonder on 09 Oct 2020
Italfarmaco receives FDA rare paediatric disease designation for givinostat in Duchenne muscular dystrophy, announces completed enrolment in EPIDYS Phase 3 trial
9 October 2020 - The Italfarmaco Group provided today an update on the development of givinostat, its proprietary histone deacetylase ...
Posted by Michael Wonder on 09 Oct 2020
BioMarin receives FDA approval of label expansion to allow maximum dose of 60 mg for Palynziq (pegvaliase-pqpz) injection for treatment of adults with PKU
7 October 2020 - New safety and efficacy data out to 3 years for first and only enzyme therapy to treat ...
Posted 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
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 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 30 Sep 2020
Simponi Aria (golimumab) approved by the U.S. FDA for active polyarticular juvenile idiopathic arthritis and extension of its active psoriatic arthritis indication in patients 2 years of age and older
30 September 2020 - Phase 3 GO-VIVA clinical trial adds to the growing body of evidence for Simponi Aria. ...
Posted by Michael Wonder on 29 Sep 2020
U.S. FDA approves Pfizer's Xeljanz (tofacitinib) for the treatment of active polyarticular course juvenile idiopathic arthritis
28 September 2020 - Pfizer announced today that the U.S. FDA approved Xeljanz (tofacitinib) for the treatment of children and adolescents ...
Posted by Michael Wonder on 28 Sep 2020
U.S. FDA approves Haegarda (C1 esterase inhibitor subcutaneous [human]) for prevention of hereditary angioedema attacks in paediatric patients
28 September 2020 - Haegarda is the first and only subcutaneous prophylactic hereditary angioedema treatment approved for children 6 years of ...