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RSS FeedPosted 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, ...
Posted by Michael Wonder on 19 Dec 2024
MAIA Biotechnology granted FDA rare paediatric disease designation for THIO as a treatment for paediatric high grade gliomas
16 December 2024 - MAIA Biotechnology today announced that the FDA has designated THIO for the treatment of pediatric-type diffuse high ...
Posted by Michael Wonder on 18 Dec 2024
Merck announces FDA acceptance of biologics license application for clesrovimab, an investigational long-acting monoclonal antibody designed to protect infants from RSV disease during their first RSV season
17 December 2024 - If approved, clesrovimab has the potential to be available to help address the burden of RSV ...
Posted by Michael Wonder on 17 Dec 2024
Johnson & Johnson submits application seeking US FDA approval of Simponi (golimumab) for the treatment of paediatric ulcerative colitis
16 December 2024 - Submission aims to expand Simponi ulcerative colitis indication to include paediatric population. ...
Posted by Michael Wonder on 17 Dec 2024
Arcutis submits Zorvye (roflumilast) cream 0.05% supplemental new drug application to the FDA for the treatment of children aged 2 to 5 with mild to moderate atopic dermatitis
16 December 2024 - Zorvye cream 0.05% provided meaningful disease clearance and rapid reduction in itch in pivotal trials. ...
Posted by Michael Wonder on 12 Dec 2024
FDA grants Revescor (rexlemestrocel-L) regenerative medicine advanced therapy designation in children with congenital heart disease
5 December 2024 - Earlier this year FDA granted Revascor both rare paediatric disease designation and orphan drug designation for ...
Posted by Michael Wonder on 07 Dec 2024
PIF Partners granted FDA rare paediatric disease designation for its proprietary small molecule in treating systemic juvenile idiopathic arthritis flares
3 December 2024 - PIF Partners announced today that the US FDA has granted rare paediatric disease designation to its proprietary ...
Posted by Michael Wonder on 06 Dec 2024
Johnson & Johnson seeks US FDA approval for first paediatric indications for Tremfya (guselkumab)
2 December 2024 - Applications filed for Tremfya to treat children with moderate to severe plaque psoriasis and active juvenile psoriatic ...
Posted by Michael Wonder on 21 Nov 2024
ImmuneSensor Therapeutics receives orphan drug and rare paediatric disease designations from the US FDA for cGAS inhibitor drug candidate, IMSB301, for the treatment of Aicardi Goutières syndrome
20 November 2024 - ImmuneSensor Therapeutics announced that the US FDA has granted both orphan drug designation and rare paediatric disease ...
Posted by Michael Wonder on 19 Nov 2024
Alkeus Pharmaceuticals receives FDA rare paediatric disease and fast track designations for gildeuretinol as a treatment for Stargardt disease
18 November 2024 - Alkeus Pharmaceuticals today announced that gildeuretinol (ALK-001), an investigational oral therapy, has received rare paediatric disease and ...
Posted by Michael Wonder on 16 Nov 2024
AlveoGene receives rare paediatric disease designation from FDA for AVG-002, its novel, inhaled gene therapy for lethal neonatal surfactant protein B deficiency
15 November 2024 - AlveoGene announces that it has been granted a rare paediatric disease designation by the US FDA ...
Posted by Michael Wonder on 14 Nov 2024
Actuate announces FDA rare paediatric disease designation granted to elraglusib for treatment of Ewing sarcoma
12 November 2024 - On-going enrolment in Phase 1/2 trial of elraglusib in relapsed/refractory Ewing sarcoma with topline Phase 1 data ...
Posted by Michael Wonder on 08 Nov 2024
Cumberland Pharmaceuticals receives FDA orphan drug and rare paediatric disease designations for new treatment of Duchenne muscular dystrophy
6 November 2024 - Cumberland Pharmaceuticals announced today that the US FDA granted orphan drug designation and rare paediatric disease ...
Posted by Michael Wonder on 08 Nov 2024
AskBio receives FDA rare paediatric disease and orphan drug designations for AB-1003 for the treatment of limb girdle muscular dystrophy type 2I/R9
7 November 2024 - First patient dosed in Phase 1/Phase 2 LION-CS101 trial of AB-1003 in August 2023, with enrolment ...