Blog
RSS FeedPosted by Michael Wonder on 31 Aug 2025
FDA approval of Leqembi subcutaneous formulation charts path to combination therapies for Alzheimer's disease
29 August 2025 - Subcutaneous delivery increases accessibility and introduces potential for at home administration, following model of diabetes and ...
Posted by Michael Wonder on 06 Jun 2024
Neurogene announces NGN-401 gene therapy for Rett syndrome selected by FDA for START pilot program
3 June 2024 - NGN-401 is one of only three CBER programs chosen by FDA. ...
Posted 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 06 Jun 2024
New cancer drug funding to be announced this year, Christopher Luxon
4 June 2024 - Prime Minister Christopher Luxon says the government is aiming to make an announcement on funding cancer ...
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
Blood cancer doctors urge Government: don’t break drug promise to our patients
6 June 2024 - More pressure has come on the Government over PHARMAC funding as an alliance of haematologists and ...
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
Ivosidenib in combination with azacitidine for the first-line treatment of patients with IDH1 R132 mutation positive acute myeloid leukaemia (final guidance)
5 June 2024 - NICE has published final evidence-based guidance on the use of ivosidenib with azacitidine in the NHS ...
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
Nivolumab for the adjuvant treatment of patients 12 years and older with completely resected melanoma at high risk of recurrence
5 June 2024 - NICE is unable to make a recommendation on the use of nivolumab (Opdivo) for the adjuvant treatment ...
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 ...