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RSS FeedPosted by Michael Wonder on 14 Sep 2024
US FDA grants RPD designation to Senhwa Biosciences silmitasertib for paediatric neuroblastoma
13 September 2024 - Senhwa Biosciences today announced that its new drug silmitasertib (CX-4945) was granted a rare paediatric disease designation ...
Posted by Michael Wonder on 13 Sep 2024
Dupixent (dupilumab) approved in the US as first and only treatment for adolescents with chronic rhinosinusitis with nasal polyps
13 September 2024 - Regeneron Pharmaceuticals and Sanofi today announced that the US FDA has approved Dupixent (dupilumab) as an add-on ...
Posted by Michael Wonder on 13 Sep 2024
Dupixent (dupilumab injection) approved in Canada for the treatment of children aged 1 year and older with eosinophilic oesophagitis
12 September 2024 - This is the ninth indication for Dupixent in Canada and its second in gastroenterology. ...
Posted by Michael Wonder on 12 Sep 2024
Empagliflozin for the treatment of adolescents with type 2 diabetes mellitus
12 September 2024 - NICE is unable to make a recommendation on the use of empagliflozin (Jardiance) in the NHS for ...
Posted by Michael Wonder on 12 Sep 2024
Ilya Pharma receives rare paediatric disease designation from the US FDA for emilimogene sigulactibac for the treatment of skin wounds in SAVI patients
11 September 2024 - Ilya Pharma today announced that it has received rare paediatric disease designation by the US FDA. ...
Posted by Michael Wonder on 11 Sep 2024
ARS Pharmaceuticals submits sNDA to FDA for neffy 1 mg dose for paediatric patients with type I allergic reactions who weigh 15 to 30 kg (33-66 lbs)
9 September 2024 - ARS Pharmaceuticals announced today the submission of a supplemental new drug application for neffy 1 mg ...
Posted by Michael Wonder on 11 Sep 2024
Health Canada approves AbbVie's Constella (linaclotide) for the treatment of functional constipation in paediatric patients 6 to 17 years of age
10 September 2024 - Submission is based on positive Phase 3 study data demonstrating linaclotide (72 mcg) resulted in increases ...
Posted by Michael Wonder on 10 Sep 2024
Cartesian Therapeutics receives FDA rare paediatric disease designation for Descartes-08 for the treatment of juvenile dermatomyositis
9 September 2024 - Cartesian Therapeutics today announced that the US FDA has granted rare paediatric disease designation to Descartes-08 ...
Posted by Michael Wonder on 08 Sep 2024
YolTech Therapeutics receives US FDA rare paediatric disease designation for YOLT-203 in treating primary hyperoxaluria type 1
4 September 2024 - YolTech Therapeutics today announced that the US FDA has granted rare paediatric disease designation to YOLT-203 for ...
Posted by Michael Wonder on 02 Sep 2024
CAMP4 Therapeutics secures rare paediatric disease designation for CMP-CPS-001 for the treatment of urea cycle disorders
27 August 2024 - CAMP4 Therapeutics today announced the US FDA has granted rare paediatric disease designation to the Company’s lead ...
Posted by Michael Wonder on 27 Aug 2024
Rhythm Pharmaceuticals announces FDA acceptance for priority review of supplemental new drug application for Imcivree (setmelanotide) in patients as young as 2 years old
26 August 2024 - FDA sets PDUFA goal date of 26 December 2024. ...
Posted by Michael Wonder on 22 Aug 2024
Opus Genetics receives rare paediatric disease designation from the US FDA for ocular gene therapy OPGx-LCA5 to treat rare inherited retinal disease LCA5
20 August 2024 - Opus Genetics today announced the US FDA has granted rare paediatric disease designation for its ocular gene ...
Posted by Michael Wonder on 13 Aug 2024
Wave Life Sciences receives FDA rare paediatric disease designation for WVE-N531 for the treatment of Duchenne muscular dystrophy
12 August 2024 - Wave Life Sciences today announced that the US FDA has granted rare paediatric disease designation to WVE-N531 ...
Posted by Michael Wonder on 12 Aug 2024
Actio Biosciences receives orphan drug and rare paediatric disease designations for the treatment of Charcot-Marie-Tooth disease 2C
8 August 2024 - Actio Biosciences today announced the US FDA has granted both orphan drug designation and rare paediatric disease ...
Posted by Michael Wonder on 11 Aug 2024
Satellos receives rare paediatric disease designation from the US FDA for SAT-3247 for the treatment of Duchenne muscular dystrophy
8 August 2024 - Satellos Bioscience announced today that the US FDA has granted rare paediatric disease designation to SAT-3247 ...