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RSS FeedPosted by Michael Wonder on 16 Aug 2023
Servier announces FDA filing acceptance and priority review for Tibsovo (ivosidenib tablets) in the treatment of IDH1 mutated relapsed or refractory myelodysplastic syndromes
15 August 2023 - Submission supported by comprehensive clinical data package, including updated results that demonstrate durable remissions and an ...
Posted by Michael Wonder on 15 Aug 2023
Lactiga receives FDA rare paediatric disease designation for groundbreaking mucosal immunoglobulin therapy for common variable immunodeficiency disease
14 August 2023 - LCTG-001 on course to be the first FDA approved biologic from human milk. ...
Posted by Michael Wonder on 07 Aug 2023
HuidaGene Therapeutics receives FDA rare paediatric disease designation for HG004 to treat inherited blindness
7 August 2023 - HG004 is a one time, direct to RPE treatment of inherited retinal disease caused by mutations in ...
Posted by Michael Wonder on 03 Aug 2023
Aardvark Therapeutics announces receipt of FDA rare paediatric disease designation for Prader-Willi syndrome
3 August 2023 - Aardvark's Phase 2 trial of oral ARD-101 in young adults with Prader-Willi syndrome is now open ...
Posted by Michael Wonder on 01 Aug 2023
FDA grants QIDP designation for Kinnear's CSA-131 drug for life-threatening Pseudomonas bacterial infections in CF
31 July 2023 - Kinnear Pharmaceuticals today announced the US FDA has granted qualified infectious disease product designation for CSA-131 ...
Posted by Michael Wonder on 20 Jul 2023
Advent Therapeutics receives FDA rare paediatric disease designation for retinol palmitate for prevention of bronchopulmonary dysplasia in premature infants
20 July 2023 - Advent plans FDA PDUFA meeting late 2023 to establish pathway for US approval of lead injectable ...
Posted by Michael Wonder on 13 Jul 2023
Aridis’ AR-301 monoclonal antibody is among the first biologics to receive FDA’s qualified infectious diseases product designation
12 July 2023 - QIDP designation for biologics provides FDA priority review status. ...
Posted by Michael Wonder on 10 Jul 2023
MyoPax receives rare paediatric disease designation from FDA for breakthrough regenerative cell product in exstrophy-epispadias complex
10 July 2023 - The US FDA granted a rare paediatric Disease designation to MyoPax.. ...
Posted by Michael Wonder on 07 Jul 2023
FDA grants rare paediatric disease designation to NS-089/NCNP-02 for the treatment of Duchenne muscular dystrophy
7 July 2023 - NS Pharma announced today the US FDA has granted rare paediatric disease designation to NS-089/NCNP-02 (brogidirsen) an ...
Posted by Michael Wonder on 06 Jul 2023
IPS Heart receives US FDA rare paediatric drug designation for ISX9-CPC stem cell therapy for treatment of cardiomyopathy associated with Danon disease
6 July 2023 - Marks third rare paediatric drug designation granted by FDA for pipeline candidate. ...
Posted by Michael Wonder on 06 Jul 2023
Astellas announces US FDA grants priority review for zolbetuximab biologics license application
6 July 2023 - Astellas Pharma today announced that the US FDA has accepted and granted priority review for the company’s ...
Posted by Michael Wonder on 05 Jul 2023
Sarepta Therapeutics announces sale of priority review voucher for $102 million
5 July 2023 - Sarepta Therapeutics today announced it had completed the sale of its rare paediatric disease priority review ...
Posted by Michael Wonder on 29 Jun 2023
Italfarmaco Group completes FDA submission of new drug application for givinostat in Duchenne muscular dystrophy and receives priority review
29 June 2023 - Submission based on completed Phase 3 program in 179 boys demonstrating significant slowing of disease progression with ...
Posted by Michael Wonder on 21 Jun 2023
bluebird bio announces FDA priority review of the biologics license application for lovotibeglogene autotemcel (lovo-cel) for patients with sickle cell disease 12 years and older with a history of vaso-occlusive events
21 June 2023 - PDUFA date set for 20 December 2023. ...
Posted by Michael Wonder on 14 Jun 2023
Eagle Pharmaceuticals receives FDA qualified infectious disease product and fast track designation for CAL02
14 June 2023 - With QIDP, Eagle expects to receive eight or ten years of regulatory exclusivity upon NDA approval. ...