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RSS FeedPosted 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 10 Dec 2024
MeiraGTx granted FDA regenerative medicine advanced therapy designation for AAV2-hAQP1 for the treatment of grade 2/3 radiation-induced xerostomia
9 December 2024 - MeiraGTx today announced that the US FDA has granted regenerative medicine advanced therapy designation to AAV2-hAQP1 for ...
Posted by Michael Wonder on 07 Dec 2024
Imfinzi granted priority review in the US for patients with muscle-invasive bladder cancer
6 December 2024 - Decision based on NIAGARA Phase 3 trial results which demonstrated a statistically significant and clinically meaningful ...
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 03 Dec 2024
Intellia Therapeutics announces FDA regenerative medicine advanced therapy designation granted to nexiguran ziclumeran for the treatment of hereditary transthyretin amyloidosis with polyneuropathy
25 November 2024 - Intellia Therapeutics today announced that the US FDA has granted regenerative medicine advanced therapy designation to nexiguran ...
Posted by Michael Wonder on 30 Nov 2024
Nurix Therapeutics receives PRIME designation from the EMA for NX-5948 for the treatment of relapsed or refractory chronic lymphocytic leukaemia
20 November 2024 - Pivotal trials of NX-5948 are planned to initiate in 2025. ...
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 ...
Posted by Michael Wonder on 06 Nov 2024
Acadia Pharmaceuticals enters into an agreement to sell its rare paediatric disease priority review voucher for $150 million
5 November 2024 - Acadia Pharmaceuticals today announced that it entered into a definitive asset purchase agreement to sell its ...
Posted by Michael Wonder on 29 Oct 2024
Allogene Therapeutics receives FDA regenerative medicine advanced therapy designation for ALLO-316, an AlloCAR T investigational product for adult patients with advanced or metastatic renal cell carcinoma
29 October 2024 - ALLO-316 was previously granted fast track designation in March 2022 by the US FDA. ...
Posted by Michael Wonder on 26 Oct 2024
FDA grants rare paediatric disease designation to Omeros’ MASP-3 inhibitor zaltenibart for treatment of C3 glomerulopathy
24 October 2024 - Phase 3 studies on the horizon in both PNH and C3G. ...
Posted by Michael Wonder on 22 Oct 2024
Papillon Therapeutics receives rare paediatric disease designation from the US FDA for PPL-002 for the treatment of Danon disease
21 October 2024 - Papillon Therapeutics today announced that the US FDA has granted rare paediatric disease designation to Papillon’s PPL-002, ...