Lisocabtagene maraleucel (liso-cel) is an investigational, autologous chimeric antigen receptor (CAR) T-cell therapy, a novel type of immunotherapy in which a patient’s own immune cells are genetically re-programmed to target and attack cancer cells. It is proposed to treat adults with relapsed or refractory large B-cell lymphoma after at least 2 prior therapies.
Emerging Technology Reports
The Emerging Technology Report provides regular updates about emerging and potentially disruptive technologies that are likely to have a significant impact in healthcare. This “early warning” report monitors new health technologies from 6 months to 2 years within a regulatory submission and tracks those as they go through clinical trial development and the regulatory approval process.
For each emerging technology, our analysts describe the technology and intended use, its prospects relative to the regulatory process and FDA information, competing technologies, relevant clinical guidelines, high-level look at the evidence such as published abstracts in the medical literature and available meeting abstracts, and available clinical trials.
Sutimlimab for Cold Agglutinin Disease
Sutimlimab is an intravenous monoclonal antibody targeting complement component 1s (C1s) for the treatment of cold agglutinin disease.
Narsoplimab for Stem Cell Transplant-Associated Thrombotic Microangiopathy
Narsoplimab is an investigational, intravenous monoclonal antibody targeting mannan-binding lectin-associated serine protease-2 (MASP-2) for the treatment of hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA).
Aducanumab for Alzheimer Disease
Aducanumab is an investigational human monoclonal anti-amyloid beta antibody in development for the treatment of Alzheimer disease.
Mavacamten for Hypertropic Cardiomyopathy
Mavacamten is an investigational oral myosin inhibitor that targets the underlying molecular mechanisms of hypertrophic cardiomyopathy (HCM). It is proposed for the treatment of patients with obstructive HCM.
Vosoritide for Achondroplasia
Vosoritide is a longer-acting C-type natriuretic peptide (CNP) analog that modulates activity of the fibroblast growth factor receptor 3 (FGFR3) by inhibiting a signaling pathway that mediates its effects, thereby stimulating endochondrial growth, resulting in enhanced growth velocity. It is proposed for the treatment of achondroplasia.
Idecabtagene Vicleucel for Multiple Myeloma
Idecabtagene vicleucel is an investigational chimeric antigen receptor (CAR) T-cell therapy targeting B-cell maturation antigen (BCMA). It is proposed for the treatment of adults with relapsed/refractory multiple myeloma who have received at least 3 prior therapies including an anti-CD38 monoclonal antibody, a proteasome inhibitor, and an immunomodulatory agent.
WiSE Cardiac Resynchronization Therapy System
The WiSE CRT system is a wireless left ventricular pacing system that works with an implanted right ventricular pacemaker and/or defibrillator for patients in whom cardiac resynchronization therapy (CRT) is indicated.
Remestemcel-L for Acute Graft-Versus-Host Disease
Remestemcel-L is an investigational allogeneic liquid cell suspension of ex-vivo cultured adult human mesenchymal stem cells derived from healthy donor bone marrow proposed for the treatment of pediatric patients with acute graft-versus-host-disease (aGVHD) refractory to standard first-line steroid therapy.
Evinacumab for Homozygous Familial Hypercholesterolemia
Evinacumab is an intravenously administered monoclonal antibody proposed for use as an adjunct to other lipid-lowering therapies in patients with homozygous familial hypercholesterolemia (HoFH).