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RSS FeedPosted by Michael Wonder on 21 Nov 2024
Arrowhead Pharmaceuticals submits new drug application to US FDA for plozasiran for the treatment of familial chylomicronemia syndrome
18 November 2024 - The new drug application is based on positive results from the Phase 3 PALISADE study. ...
Posted by Michael Wonder on 21 Nov 2024
ImmuneSensor Therapeutics receives orphan drug and rare paediatric disease designations from the US FDA for cGAS inhibitor drug candidate, IMSB301, for the treatment of Aicardi Goutières syndrome
20 November 2024 - ImmuneSensor Therapeutics announced that the US FDA has granted both orphan drug designation and rare paediatric disease ...
Posted by Michael Wonder on 19 Nov 2024
FDA grants breakthrough therapy designation to Huahui Health's libevitug (HH-003) for treatment of chronic hepatitis D virus infection
18 November 2024 - Huahui Health today announced that its investigational hepatitis D and B drug candidate, Libevitug (HH-003), an ...
Posted by Michael Wonder on 19 Nov 2024
Alkeus Pharmaceuticals receives FDA rare paediatric disease and fast track designations for gildeuretinol as a treatment for Stargardt disease
18 November 2024 - Alkeus Pharmaceuticals today announced that gildeuretinol (ALK-001), an investigational oral therapy, has received rare paediatric disease and ...
Posted by Michael Wonder on 18 Nov 2024
Vaderis receives FDA fast track designation for VAD044 for the treatment of hereditary haemorrhagic telangiectasia
18 November 2024 - Vaderis Therapeutics today announces that the US FDA has designated the allosteric AKT inhibitor VAD044 a fast ...
Posted by Michael Wonder on 18 Nov 2024
Alentis receives FDA fast track designation for ALE.P02 for the treatment of CLDN1 positive squamous solid tumours
18 November 2024 - ALE.P02 is a first in class antibody-drug conjugate targeting CLDN1. ...
Posted by Michael Wonder on 17 Nov 2024
Azurity Pharmaceuticals announces FDA approval of Danziten (nilotinib) tablets, the first and only nilotinib with no mealtime restrictions
14 November 2024 - Azurity Pharmaceuticals announced today that the US FDA has approved Danziten, the first and only nilotinib with ...
Posted by Michael Wonder on 16 Nov 2024
AlveoGene receives rare paediatric disease designation from FDA for AVG-002, its novel, inhaled gene therapy for lethal neonatal surfactant protein B deficiency
15 November 2024 - AlveoGene announces that it has been granted a rare paediatric disease designation by the US FDA ...
Posted by Michael Wonder on 15 Nov 2024
FDA approves revumenib for relapsed or refractory acute leukaemia with a KMT2A translocation
15 November 2024 - Today, the FDA approved revumenib (Revuforj, Syndax Pharmaceuticals), a menin inhibitor, for relapsed or refractory acute ...
Posted by Michael Wonder on 15 Nov 2024
Dupixent (dupilumab) sBLA accepted for FDA review for the treatment of chronic spontaneous urticaria
15 November 2024 - Resubmission includes new pivotal data which confirm Dupixent significantly reduced itch and hive activity. ...
Posted by Michael Wonder on 14 Nov 2024
Actuate announces FDA rare paediatric disease designation granted to elraglusib for treatment of Ewing sarcoma
12 November 2024 - On-going enrolment in Phase 1/2 trial of elraglusib in relapsed/refractory Ewing sarcoma with topline Phase 1 data ...
Posted by Michael Wonder on 14 Nov 2024
PTC Therapeutics announces FDA approval of AADC deficiency gene therapy
13 November 2024 - PTC Therapeutics announced today the US FDA accelerated approval of its gene therapy for the treatment of ...
Posted by Michael Wonder on 12 Nov 2024
Abeona Therapeutics announces FDA acceptance of BLA resubmission of prademagene zamikeracel for the treatment of recessive dystrophic epidermolysis bullosa
12 November 2024 - Abeona Therapeutics today announced that the US FDA has accepted for review Abeona’s resubmission of its ...
Posted by Michael Wonder on 12 Nov 2024
Genascence granted FDA fast track designation for GNSC-001 in patients with osteoarthritis of the knee
12 November 2024 - GNSC-001 is designed to offer long-term, sustained inhibition of IL-1 following a single injection into the affected ...
Posted by Michael Wonder on 12 Nov 2024
Intercept receives complete response letter from FDA addressing Ocaliva supplemental new drug application
13 November 2024 - Intercept Pharmaceuticals today announced that the US FDA has issued a complete response letter that addresses ...