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RSS FeedPosted by Michael Wonder on 22 Apr 2021
Allogene Therapeutics announces FDA regenerative medicine advanced therapy designation granted to ALLO-715, an AlloCAR T cell therapy in development for relapsed/refractory multiple myeloma
21 April 2021 - Regenerative medicine advanced therapy designation follows proof of concept data from ALLO-715 UNIVERSAL trial in heavily pretreated, ...
Posted by Michael Wonder on 15 Apr 2021
FibroGen receives rare paediatric disease designation from the U.S. FDA for pamrevlumab for the treatment of Duchenne muscular dystrophy
15 April 2021 - FibroGen announced that the U.S. FDA has granted rare paediatric disease designation for the company’s anti-CTGF antibody, ...
Posted by Michael Wonder on 14 Apr 2021
Moleculin awarded new rare paediatric disease designation from U.S. FDA for WP1066 for the treatment of ependymoma
14 April 2021 - Moleculin Biotech announced today that the U.S. FDA has granted rare paediatric disease designation to its ...
Posted by Michael Wonder on 29 Mar 2021
Mirum Pharmaceuticals announces FDA acceptance of new drug application and priority review for maralixibat in Alagille syndrome
29 March 2021 - PDUFA action date is 29 September 2021. ...
Posted by Michael Wonder on 16 Mar 2021
Merck receives priority review from FDA for new drug application for HIF-2α inhibitor belzutifan (MK-6482)
16 March 2021 - Application based on objective response rate from Phase 2 trial evaluating belzutifan in patients with von Hippel-Lindau ...
Posted by Michael Wonder on 09 Mar 2021
Rocket Pharmaceuticals receives FDA regenerative medicine advanced therapy designation for RP-L201 gene therapy for leukocyte adhesion deficiency-I
9 March 2021 - Enrollment complete in Phase 1/2 clinical trial of RP-L201 for the treatment of LAD-I. ...
Posted by Michael Wonder on 08 Mar 2021
BioMarin announces FDA regenerative medicine advanced therapy designation granted to valoctocogene roxaparvovec, investigational gene therapy for haemophilia A
8 March 2021 - RMAT designation granted by FDA during bleeding disorders awareness month. ...
Posted by Michael Wonder on 08 Mar 2021
Cara Therapeutics and Vifor Pharma announce U.S. FDA acceptance and priority review of NDA for Korsuva injection in haemodialysis patients with moderate to severe pruritus
8 March 2021 - FDA has set Prescription Drug User Fee Act target action date of 23 August 2021. ...
Posted by Michael Wonder on 03 Mar 2021
On Target Laboratories announces U.S. FDA acceptance and priority review of new drug application for pafolacianine sodium injection for identification of ovarian cancer during surgery
3 March 2021 - On Target Laboratories today announced that the U.S. FDA has accepted its new drug application for priority ...
Posted by Michael Wonder on 26 Feb 2021
Retrotope granted rare paediatric disease designation from FDA for lead development candidate, RT001, in two life-threatening neurodegenerative indications
25 February 2021 - RT001 also granted fast track designation by FDA in Friedreich’s ataxia. ...
Posted by Michael Wonder on 24 Feb 2021
U.S. FDA accepts for priority review Pfizer's application for Ticovac (tick-borne encephalitis vaccine)
23 February 2021 - If approved, the vaccine may help reduce the risk of tick-borne encephalitis for people traveling to endemic ...
Posted by Michael Wonder on 17 Feb 2021
FDA grants sotorasib priority review designation for the treatment of patients with KRAS G12C mutated locally advanced or metastatic non-small cell lung cancer
16 February 2021 - FDA target action date is 16 August 2021 ...
Posted by Michael Wonder on 16 Feb 2021
Forge Biologics receives FDA fast track, orphan drug, and rare paediatric disease designations for FBX-101 gene therapy for patients with Krabbe disease
16 February 2021 - FBX-101 is a first-in-human gene therapy utilising an adeno-associated virus to deliver a functioning copy of the ...
Posted by Michael Wonder on 16 Feb 2021
Sesen Bio announces FDA acceptance and priority review of its biologics license application for Vicineum
16 February 2021 - FDA stated it is not currently planning to hold an advisory committee meeting. ...
Posted by Michael Wonder on 15 Feb 2021
Polaryx Therapeutics receives both rare paediatric disease and orphan drug designations for the treatment of Krabbe disease with PLX-300
10 February 2021 - Polaryx Therapeutics announced today that it has received both rare paediatric disease and orphan drug designations for ...