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RSS FeedPosted by Michael Wonder on 05 Jun 2024
uniQure receives FDA regenerative medicine advanced therapy designation for investigational gene therapy AMT-130 in Huntington’s disease
3 June 2024 - Designation based on 24 month interim Phase 1/2 clinical data for AMT-130 announced in December 2023. ...
Posted by Michael Wonder on 03 Jun 2024
Zanidatamab granted priority review for HER2 positive metastatic biliary tract cancer
29 May 2024 - Target action (PDUFA) date set for 29 November 2024. ...
Posted by Michael Wonder on 30 May 2024
FDA grants priority review to Roche’s inavolisib for advanced hormone receptor positive, HER2 negative breast cancer with a PIK3CA mutation
29 May 2024 - The target action date for the FDA decision is 27 November 2024. ...
Posted by Michael Wonder on 29 May 2024
FDA grants priority review to Merck’s application for Keytruda (pembrolizumab) plus chemotherapy as first-line treatment of patients with unresectable advanced or metastatic malignant pleural mesothelioma
29 May 2024 - Acceptance based on results from the pivotal Phase 3 CCTG IND.227/KEYNOTE-483 trial, which demonstrated a statistically significant ...
Posted by Michael Wonder on 23 May 2024
Cartesian Therapeutics receives FDA regenerative medicine advanced therapy designation for Descartes-08 for the treatment of myasthenia gravis
22 May 2024 - Cartesian Therapeutics today announced that the US FDA has granted regenerative medicine advanced therapy designation for ...
Posted by Michael Wonder on 21 May 2024
Wugen announces RMAT and PRIME designations for WU-CART-007 to accelerate regulatory reviews
20 May 2024 - Researchers to present new Phase 2 data showing safety and anti-leukaemic activity with WU-CART-007 and additional new ...
Posted 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 ...