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RSS FeedPosted by Michael Wonder on 06 Jun 2024
Denali Therapeutics announces FDA has selected DNL126 for MPS IIIA (Sanfilippo syndrome type A) for START pilot program intended to accelerate development of rare disease therapies
3 June 2024 - START is a pilot program newly launched jointly by the FDA CDER and CBER divisions to further ...
Posted by Michael Wonder on 06 Jun 2024
Moderna's investigational therapeutic for methylmalonic acidaemia (mRNA-3705) selected by US FDA for START pilot program
6 June 2024 - Center for Biologics Evaluation and Research has chosen mRNA-3705 as one of four investigational medicines for ...
Posted by Michael Wonder on 06 Jun 2024
FDA accepts for review treosulfan NDA resubmission
6 June 2024 - Today, Medexus Pharmaceuticals was informed by medac, licensor of Medexus's commercialisation rights to treosulfan, that the ...
Posted by Michael Wonder on 05 Jun 2024
Catalyst Pharmaceuticals receives US FDA approval for increased maximum daily dose for Firdapse
30 May 2024 - Catalyst Pharmaceuticals today announced that the US FDA has approved its supplemental new drug application increasing the ...
Posted by Michael Wonder on 05 Jun 2024
Why should the FDA focus on pragmatic clinical research?
3 June 2024 - Traditional randomised clinical trials have long been a key tool underpinning drug and device development. ...
Posted by Michael Wonder on 05 Jun 2024
ICER publishes protocol for annual assessment of drug coverage policies that present barriers to fair access
5 June 2024 - This year’s analysis will include the top ten commercial formularies by covered lives, and include drugs ...
Posted by Michael Wonder on 05 Jun 2024
Adicet Bio receives FDA fast track designation for ADI-001 in lupus nephritis
5 June 2024 - Adicet Bio today announced the US FDA has granted fast track designation to ADI-001 for the potential ...
Posted by Michael Wonder on 05 Jun 2024
TScan Therapeutics receives FDA’s regenerative medicine advanced therapy designation for its two lead TCR-T therapy candidates for the treatment of heme malignancies
29 May 2024 - RMAT designation granted for both TSC-100 and TSC-101 for the treatment of patients with AML, ALL, and ...
Posted 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 05 Jun 2024
Johnson & Johnson submits supplemental new drug application to US FDA seeking expanded paediatric indication for HIV-1 therapy Prezcobix
4 June 2024 - Johnson & Johnson today announced the submission of a supplemental new drug application to the US FDA ...
Posted by Michael Wonder on 05 Jun 2024
Quince Therapeutics receives US FDA fast track designation for EryDex system
3 June 2024 - Quince Therapeutics today announced that the US FDA has granted fast track designation for its EryDex System ...
Posted by Michael Wonder on 05 Jun 2024
Telix completes TLX250-CDx (Zircaix) BLA submission for kidney cancer imaging
3 June 2024 - Telix today announces that it has completed the submission of a biologics license application to the ...
Posted by Michael Wonder on 05 Jun 2024
Rinvoq (upadacitinib) now available for paediatric patients two years and older with polyarticular juvenile idiopathic arthritis and psoriatic arthritis
4 June 2024 - First indications of Rinvoq (upadacitinib) for paediatric patients two years of age and older. ...
Posted by Michael Wonder on 04 Jun 2024
Iterum Therapeutics receives FDA acceptance of resubmission of NDA for oral sulopenem for the treatment of uncomplicated urinary tract infections
31 May 2024 - Oral sulopenem NDA has been assigned a PDUFA action date of 25 October 2024. ...
Posted by Michael Wonder on 04 Jun 2024
Repare Therapeutics announces fast track designation granted by the FDA for lunresertib in combination with camonsertib for the treatment of platinum resistant ovarian cancer
4 June 2024 - Repare Therapeutics today announced the US FDA has granted fast track designation to lunresertib in combination with ...