Blog
RSS FeedPosted by Michael Wonder on 15 May 2024
PTC Therapeutics announces FDA acceptance and priority review of the BLA for Upstaza
14 May 2024 - PDUFA target action date of 13 November 2024. ...
Posted by Michael Wonder on 13 May 2024
Dupixent (dupilumab) sBLA accepted for FDA priority review for treatment of adolescents with chronic rhinosinusitis with nasal polyposis
13 May 2024 - If approved, Dupixent would be the first treatment in the US indicated for adolescents aged 12-17 years ...
Posted by Michael Wonder on 06 May 2024
Merus announces US FDA acceptance and priority review of biologics license application for zenocutuzumab for the treatment of NRG1 positive NSCLC and PDAC
6 May 2024 - If approved, zenocutuzumab will be the first targeted therapy for NRG1 positive cancer. ...
Posted by Michael Wonder on 06 May 2024
Taysha Gene Therapies announces regenerative medicine advanced therapy designation granted by US FDA for TSHA-102 in Rett syndrome
2 May 2024 - RMAT designation follows FDA’s review of available safety and efficacy data from the first three patients dosed ...
Posted by Michael Wonder on 02 Apr 2024
Solid Biosciences receives rare paediatric disease designation from the FDA for Duchenne muscular dystrophy gene therapy candidate SGT-003
1 April 2024 - Site initiations scheduled for April; patient dosing expected to begin in Q2 ,2024. ...
Posted by Michael Wonder on 27 Mar 2024
Syndax announces FDA priority review of NDA for revumenib for the treatment of relapsed/refractory KMT2Ar acute leukaemia
26 March 2024 - PDUFA action date set for 26 September 2024. ...
Posted by Michael Wonder on 26 Mar 2024
Tonix Pharmaceuticals receives rare paediatric disease designation from the FDA for TNX-2900 for the treatment of Prader-Willi syndrome
25 March 2024 - TNX-2900 is a proprietary magnesium-potentiated formulation of intranasal oxytocin, a naturally occurring hormone that reduces appetite and ...
Posted by Michael Wonder on 25 Mar 2024
FDA grants Argenica rare paediatric disease designation for ARG-007 in the treatment of HIE
25 March 2024 - Argenica Therapeutics is pleased to announce the US FDA has granted its neuroprotective drug ARG-007 rare ...
Posted by Michael Wonder on 22 Mar 2024
Lisata Therapeutics announces US FDA rare paediatric disease designation granted to LSTA1 for the treatment of osteosarcoma
21 March 2024 - Lisata Therapeutics today announced that the US FDA has granted rare paediatric disease designation to LSTA1, the ...
Posted by Michael Wonder on 19 Mar 2024
Deverra Therapeutics granted FDA regenerative medicine advanced therapy and orphan drug designations for DVX101 (Dilanubicel) for the treatment of acute myeloid leukaemia
19 March 2024 - Deverra Therapeutics today announced that the US FDA granted regenerative medicine advanced therapy designation to its lead ...
Posted by Michael Wonder on 14 Mar 2024
PepGen receives US FDA orphan drug and rare paediatric disease designations for PGN-EDO51 for the treatment of Duchenne muscular dystrophy
13 March 2024 - PepGen today announced that the US FDA granted both orphan drug and rare paediatric disease designations for ...
Posted by Michael Wonder on 28 Feb 2024
Applied Therapeutics announces FDA acceptance and priority review of new drug application for govorestat for the treatment of classic galactosaemia
28 February 2024 - PDUFA target action date of 28 August 2024. ...
Posted by Michael Wonder on 28 Feb 2024
Incyte announces US FDA grants priority review for axatilimab for the treatment of chronic graft versus host disease
27 February 2024 - Priority review acceptance based on positive results of AGAVE-201 study. ...
Posted by Michael Wonder on 27 Feb 2024
US FDA accepts for priority review the supplemental biologics license application for epcoritamab (Epkinly) for difficult to treat relapsed or refractory follicular lymphoma
26 February 2024 - FDA grants priority review with target action date of 28 June 2024. ...
Posted by Michael Wonder on 23 Feb 2024
Dupixent (dupilumab) sBLA accepted for FDA priority review for treatment of COPD with type 2 inflammation
23 February 2024 - Priority review granted based on positive results from two Phase 3 trials; if approved, Dupixent would be ...