Blog
RSS FeedPosted by Michael Wonder on 03 Oct 2024
Calquence granted priority review in the US for patients with untreated mantle cell lymphoma
3 October 2024 - Submission to be reviewed under Project Orbis. ...
Posted by Michael Wonder on 01 Oct 2024
FDA grants rare paediatric disease designation to MDL-101 for the treatment of congenital muscular dystrophy type 1a
30 September 2024 - Modalis Therapeutics today announced that the US FDA has granted rare paediatric disease designation to MDL-101, a ...
Posted by Michael Wonder on 01 Oct 2024
Enhertu granted priority review in the US for patients with HER2 low or HER2 ultra-low metastatic breast cancer who have received at least one line of endocrine therapy
1 October 2024 - Based on DESTINY-Breast06 phase 3 trial which demonstrated a statistically significant and clinically meaningful progression-free survival ...
Posted by Michael Wonder on 29 Sep 2024
Enterprise Therapeutics granted rare paediatric disease designation in the US for novel cystic fibrosis investigational therapy ETD001
26 September 2024 - ETD001 is a novel, first in class blocker of the epithelial sodium channel aimed at treating people ...
Posted by Michael Wonder on 24 Sep 2024
Lantern Pharma announces three US FDA rare paediatric disease designations granted to LP-184 in multiple ultra rare children’s cancers
23 September 2024 - Lantern Pharma announced today that the company has been granted three rare paediatric disease designations by the ...
Posted by Michael Wonder on 21 Sep 2024
Voucher program notches win as clock ticks
20 September 2024 - The FDA’s priority review voucher program for rare paediatric diseases is set to lapse on 30 September ...
Posted by Michael Wonder on 17 Sep 2024
Poseida Therapeutics receives regenerative medicine advanced therapy designation from FDA for P-BCMA-ALLO1 to treat relapsed/refractory multiple myeloma
16 September 2024 - FDA evaluated RMAT application based on positive clinical data from ongoing Phase 1 study of P-BCMA-ALLO1. ...
Posted by Michael Wonder on 14 Sep 2024
US FDA grants RPD designation to Senhwa Biosciences silmitasertib for paediatric neuroblastoma
13 September 2024 - Senhwa Biosciences today announced that its new drug silmitasertib (CX-4945) was granted a rare paediatric disease designation ...
Posted by Michael Wonder on 12 Sep 2024
Ilya Pharma receives rare paediatric disease designation from the US FDA for emilimogene sigulactibac for the treatment of skin wounds in SAVI patients
11 September 2024 - Ilya Pharma today announced that it has received rare paediatric disease designation by the US FDA. ...
Posted by Michael Wonder on 10 Sep 2024
BridgeBio receives FDA’s regenerative medicine advanced therapy designation for BBP-812 Canavan disease gene therapy program
10 September 2024 - Receipt of RMAT designation is based on preliminary clinical evidence from the CANaspire Phase 1/2 clinical trial, ...
Posted by Michael Wonder on 10 Sep 2024
Cartesian Therapeutics receives FDA rare paediatric disease designation for Descartes-08 for the treatment of juvenile dermatomyositis
9 September 2024 - Cartesian Therapeutics today announced that the US FDA has granted rare paediatric disease designation to Descartes-08 ...
Posted by Michael Wonder on 08 Sep 2024
YolTech Therapeutics receives US FDA rare paediatric disease designation for YOLT-203 in treating primary hyperoxaluria type 1
4 September 2024 - YolTech Therapeutics today announced that the US FDA has granted rare paediatric disease designation to YOLT-203 for ...
Posted by Michael Wonder on 03 Sep 2024
Obsidian Therapeutics receives FDA regenerative medicine advanced therapy designation for OBX-115 for the treatment of advanced melanoma
3 September 2024 - Obsidian Therapeutics today announced that the US FDA has granted regenerative medicine advanced therapy designation to ...
Posted by Michael Wonder on 02 Sep 2024
CAMP4 Therapeutics secures rare paediatric disease designation for CMP-CPS-001 for the treatment of urea cycle disorders
27 August 2024 - CAMP4 Therapeutics today announced the US FDA has granted rare paediatric disease designation to the Company’s lead ...
Posted by Michael Wonder on 01 Sep 2024
Ipsen announces sale of priority review voucher for $158 million
27 August 2024 - Ipsen has entered into an agreement to sell its rare paediatric disease priority review voucher to ...