Blog
RSS FeedPosted by Michael Wonder on 20 Aug 2020
Polaryx Therapeutics receives FDA fast track designation to PLX-200 for the treatment of patients with juvenile neuronal ceroid lipofuscinosis
20 August 2020 - Polaryx Therapeutics announced today that the U.S. FDA has granted fast track designation to PLX-200 for ...
Posted by Michael Wonder on 12 Aug 2020
Theranexus and BBDF obtain orphan drug designation and rare paediatric disease designation from the FDA for BBDF-101 for Batten disease
11 August 2020 - Theranexus, a biopharmaceutical company innovating in the treatment of neurological diseases, and Beyond Batten Disease Foundation ...
Posted by Michael Wonder on 08 Aug 2020
U.S. Food and Drug Administration approves Lampit (nifurtimox) for the treatment of Chagas disease in children
7 August 2020 - Only Chagas disease treatment approved in U.S. for use in children from birth to less than 18 ...
Posted by Michael Wonder on 07 Aug 2020
US FDA grants rare paediatric disease designation to paxalisib for DIPG
7 August 2020 - Kazia Therapeutics is pleased to announce that the United States FDA has awarded rare paediatric disease designation ...
Posted by Michael Wonder on 05 Aug 2020
Cerecor receives rare paediatric disease designation for CERC-006 in lymphatic malformations
4 August 2020 - Cerecor today announced that the U.S. FDA granted rare paediatric disease designation to CERC-006, a dual inhibitor ...
Posted by Michael Wonder on 04 Aug 2020
Pacira BioSciences announces FDA acceptance of sNDA for Exparel use in paediatric patients
4 August 2020 - sNDA submission based on positive Phase 3 data supporting expansion of the Exparel label to include use ...
Posted by Michael Wonder on 31 Jul 2020
U.S. Food and Drug Administration approves Stelara (ustekinumab) for treatment of paediatric patients with moderate to severe plaque psoriasis
30 July 2020 - Stelara is the first and only biologic to target interleukin (IL)-12 and IL-23 approved for paediatric psoriasis ...
Posted by Michael Wonder on 30 Jul 2020
Marinus Pharmaceuticals receives rare paediatric disease designation from FDA for ganaxolone for the treatment of CDKL5 deficiency disorder
30 July 2020 - Marinus Pharmaceuticals announced today that the U.S. FDA has granted rare paediatric disease designation for the ...
Posted by Michael Wonder on 29 Jul 2020
Protara Therapeutics receives rare paediatric disease designation for TARA-002 for the treatment of lymphatic malformations
28 July 2020 - Protara Therapeutics today announced that the U.S. FDA has granted rare paediatric disease designation for TARA-002 for ...
Posted by Michael Wonder on 28 Jul 2020
HemoShear Therapeutics receives FDA fast track and rare paediatric disease designations for HST5040 to treat methylmalonic acidaemia and propionic acidaemia
28 July 2020 - HemoShear Therapeutics has received fast track and rare paediatric disease designations from the U.S. FDA for ...
Posted by Michael Wonder on 28 Jul 2020
Momenta Pharmaceuticals announces FDA rare paediatric disease designation for nipocalimab in HDFN
28 July 2020 - Momenta Pharmaceuticals today announced that its novel drug candidate, nipocalimab, has received rare paediatric disease designation ...
Posted by Michael Wonder on 16 Jul 2020
Knopp Biosciences receives rare paediatric disease designation for Kv7 activator KB-3061 for treatment of KCNQ2 epileptic encephalopathy
15 July 2020 - Knopp Biosciences announced today that it has received rare paediatric disease designation from the U.S. FDA ...
Posted by Michael Wonder on 13 Jul 2020
Ipsen announces updated indication for Dysport (abobotulinumtoxinA) for the treatment of spasticity in children
10 July 2020 - Dysport is now FDA approved to treat both upper and lower limb spasticity in paediatric patients two ...
Posted by Michael Wonder on 10 Jul 2020
FDA approves expanded Botox (onabotulinumtoxinA) label for the treatment of paediatric patients with spasticity
9 July 2020 - Updated label now includes paediatric patients with lower limb spasticity caused by cerebral palsy. ...
Posted by Michael Wonder on 07 Jul 2020
Senhwa Biosciences's silmitasertib receives rare paediatric disease designation from U.S. FDA for treatment of recurrent sonic hedgehog medulloblastoma
7 July 2020 - Senhwa Biosciences today announced that the U.S. FDA granted rare paediatric disease designation for its drug silmitasertib, ...