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Novel Drugs & Emergent Therapeutics Archive 2023

 
Date Indication Company Drug / Device Status Title  & Link
15/11/23

 		 Paediatrics X-linked myotubular myopathy (XLMTM)


 		 Astellis


 		 AT132 (resamirigene bilparvovec)

 		 AT132 IND and ASPIRO trial currently on clinical hold.

 		 Astellis Pharma published preliminary results from  APPIRO trial regarding safety and efficacy of investigational AT132 (resamirigene bilparvovec), an adeno-associated viral vector (AAV) gene replacement therapy designed to deliver a functional human MTM1 gene for the treatment of pediatric patients with X-linked myotubular myopathy (XLMTM).
ClinicalTrials.gov ID NCT03199469
09/11/23 Congenital thrombotic thrombocytopenic purpura (cTTP) Takeda Adzynma Approved FDA approves first recombinant protein product indicated for prophylactic (preventive) or on demand enzyme replacement therapy (ERT) in adult and pediatric patients with congenital thrombotic thrombocytopenic purpura
FDA Docket FDA-2014-D-1461
07/11/23 Sjogren's Syndrom Amgen Dazodalebep Phase 2 Complete Amgen presents new data from Phase 2 Trial of Dazodalebep in Sjogren's Syndrome
Phase 2 study
ClinicalTrials.gov. ID NCT04129164
20/09/23 Allergy, Anaphylaxis ARS Pharma Neffy Approval Declined US FDA declines to approve first nasal spray equivalent of EpiPen
14/07/23 Graft-vs-Host Disease Syndax, Incyte Axatilimab Complete Syndax and Incyte Announce Positive Topline Results from the Pivotal AGAVE-201 Trial of Axatilimab in Chronic Graft-Versus-Host Disease
Phase 2 Study, 
ClinicalTrials.gov ID NCT04710576
19/09/23 Atopic Dermatitis Arcutis Roflumilast Complete Roflumilast Cream Effective in Pediatric Atopic Dermatitis Trial
Clinicaltrials.gov ID NCT04845620
28/07/23  Telemedicine, Respiratory Devices U. Washington Telemedicine, Monitoring  In trial
Smartphone system developed to measure oxygenation levels
24/07/23 HABP, VABP Entasis Therapeutics Xacdura Approved FDA grants approval for Xacdura for use in the treatment of ventilator-associated bacterial pneumonia
23/02/23 Haemophilia Sanofi Altuviiio Approved FDA approves Sanofi’s long-lasting hemophilia drug
18/11/22 Diabetes Provention Bio Tzield Approved Tzield: FDA approves first drug to delay type 1 diabetes
18/08/22 Beta-Thalassemia BlueBird Bio Zynteglo Approved Zynteglo Approved as First Gene Therapy for Beta-Thalassemia
18/03/22 Rare Diseases, CDKL5 Epilepsy Marinus ZTALMY® (ganaxolone) Approved Marinus Pharmaceuticals Announces FDA Approval of ZTALMY® (ganaxolone) for CDKL5 Deficiency Disorder.
 
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