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RSS FeedPosted 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. ...
Posted by Michael Wonder on 12 Jun 2023
Capivasertib in combination with Faslodex granted priority review in the US for patients with advanced hormone receptor positive breast cancer
12 June 2023 - Decision based on CAPItello-291 Phase 3 trial results which showed the combination reduced the risk of ...
Posted by Michael Wonder on 02 Jun 2023
Pharming announces sale of priority review voucher
1 June 2023 - Pharming announces that it has entered into a definitive agreement with Novartis to sell its rare ...
Posted by Michael Wonder on 27 May 2023
Iovance Biotherapeutics announces US FDA acceptance of the biologics license application of lifileucel for the treatment of advanced melanoma
26 May 2023 - Priority review granted with PDUFA action date of 25 November 2023. ...
Posted by Michael Wonder on 26 May 2023
Takeda and HUTCHMED announce new drug application for fruquintinib for treatment of previously treated metastatic colorectal cancer granted priority review
25 May 2023 - PDUFA target action date set for 30 November 2023. ...
Posted by Michael Wonder on 23 May 2023
RegenxBio receives FDA regenerative medicine advanced therapy designation for RGX-121 gene therapy for Hunter syndrome
23 May 2023 - CAMPSIITE trial is enrolling mucopolysaccharidosis type II patients as part of a pivotal program that incorporates ...
Posted by Michael Wonder on 23 May 2023
Rocket Pharmaceuticals receives FDA regenerative medicine advanced therapy designation for RP-L301 gene therapy for pyruvate kinase deficiency
23 May 2023 - Rocket Pharmaceuticals today announced that the US FDA has granted regenerative medicine advanced therapy designation to ...
Posted by Michael Wonder on 17 May 2023
US FDA grants priority review of TAK-755 for the treatment of congenital thrombotic thrombocytopenic purpura
16 May 2023 - FDA has granted TAK-755 priority review and rare paediatric disease designation, as well as fast track designation ...
Posted by Michael Wonder on 16 May 2023
FDA grants SiSaf’s innovative siRNA therapy SIS-101-ADO orphan drug designation and rare paediatric disease designation for the treatment of autosomal dominant osteopetrosis
15 May 2023 - SiSaf announces that SIS-101-ADO, its siRNA therapeutic, has been granted orphan drug designation and rare paediatric disease ...