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RSS FeedPosted by Michael Wonder on 23 May 2025
Relief Therapeutics receives FDA rare paediatric disease designation for RLF-TD011
22 May 2025 - Relief Therapeutics today announced that the US FDA has granted rare paediatric disease designation to RLF-TD011 ...
Posted by Michael Wonder on 21 May 2025
US FDA grants approval for Jivi anti-hemophilic factor (recombinant), PEGylated-aucl in paediatric patients 7 to under 12 years of age with haemophilia A
19 May 2025 - Bayer announced that the US FDA has approved Jivi, a recombinant DNA-derived, extended half-life factor VIII ...
Posted by Michael Wonder on 16 May 2025
Kaerus Bioscience's lead candidate KER-0193 granted orphan drug designation and rare paediatric drug designation by US FDA for treatment of Fragile X syndrome
14 May 2025 - Kaerus Bioscience today announces that its lead candidate KER-0193 has been granted both orphan drug designation and ...
Posted by Michael Wonder on 16 May 2025
FDA grants regenerative medicine advanced therapy designation for BrainChild Bio’s B7-H3 CAR T-cell therapy for incurable paediatric brain tumours
15 May 2025 - BrainChild Bio advancing BCB-276 towards BLA submission with a single pivotal Phase 2 trial planned to ...
Posted by Michael Wonder on 15 May 2025
BioCryst announces FDA acceptance of NDA for Orladeyo (berotralstat) oral granules in patients with hereditary angioedema aged 2 to 11 years
14 May 2025 - FDA grants priority review of application, with PDUFA target action date of 12 September 2025. ...
Posted by Michael Wonder on 29 Apr 2025
Innorna announces FDA rare paediatric disease designation granted to IN013 for treatment of Wilson disease
25 February 2025 - Innorna is thrilled to announce that the US FDA has granted rare paediatric disease designation to ...
Posted by Michael Wonder on 17 Apr 2025
Neurelis announces FDA approval for immediate use seizure medication Valtoco (diazepam nasal spray) in age 2 to 5
16 April 2025 - Valtoco maintains orphan drug exclusivity for Valtoco to treat episodes of frequent seizures. ...
Posted by Michael Wonder on 08 Apr 2025
Teva announces FDA filing acceptance for Ajovy (fremanezumab) in paediatric episodic migraine prevention
7 April 2025 - If approved for an expanded paediatric indication, Ajovy would be the first calcitonin gene-related peptide antagonist for ...
Posted by Michael Wonder on 12 Mar 2025
Atsena Therapeutics granted US FDA fast track designation for ATSN-201 gene therapy to treat X-linked retinoschisis
12 March 2025 - Marks third FDA designation for ATSN-201, which has also received rare paediatric disease designation and orphan drug ...
Posted by Michael Wonder on 12 Mar 2025
Sydnexis announces FDA acceptance of new drug application and PDUFA date for SYD-101 for the treatment of progression of paediatric myopia
11 March 2025 - Sydnexis today announced that the US FDA has accepted its new drug application for SYD-101 and ...
Posted by Michael Wonder on 28 Feb 2025
FDA approves Odactra for the treatment of house dust mite allergy in young children
27 February 2025 - ALK today announced that the US FDA has approved ALK’s Odactra tablet for use in young children ...
Posted by Michael Wonder on 10 Jan 2025
ViGeneron announces FDA rare paediatric disease designation for VG901
8 January 2025 - ViGeneron today announced two important milestones for its novel gene therapy candidate VG901, to treat patients ...
Posted by Michael Wonder on 09 Jan 2025
Denali Therapeutics announces US FDA breakthrough therapy designation granted to tividenofusp alfa for the treatment of Hunter syndrome
8 January 2025 - Denali Therapeutics today announced that the US FDA has granted breakthrough therapy designation for tividenofusp alfa (DNL310) ...
Posted by Michael Wonder on 26 Dec 2024
Rhythm Pharmaceuticals announces FDA approval of Imcivree (setmelanotide) for patients as young as 2 years old
20 December 2024 - Rhythm Pharmaceuticals today announced that the US FDA has approved an expanded indication for Imcivree (setmelanotide) to ...
Posted by Michael Wonder on 19 Dec 2024
FDA approves remestemcel-L-rknd for steroid-refractory acute graft versus host disease in paediatric patients
18 December 2024 - Today, the FDA approved remestemcel-L-rknd (Ryoncil, Mesoblast), an allogeneic bone marrow derived mesenchymal stromal cell therapy, ...