Casimersen is an investigational intravenously administered antisense oligonucleotide proposed for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 45 skipping.
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.
Berotralstat for Hereditary Angioedema
Berotralstat is an oral plasma kallikrein inhibitor for prevention of hereditary angioedema attacks.
Retevmo (Selpercatinib) for Non-Small Cell Lung Cancer (NSCLC)
Retevmo (selpercatinib) is a novel oral highly selective RET inhibitor indicated for the treatment of adult patients with metastatic RET fusion-positive non-small cell lung cancer (NSCLC).
Lisocabtagene Maraleucel for Large B-Cell Lymphoma
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.
Voclosporin for Lupus Nephritis
Voclosporin is an oral, investigational calcineurin inhibitor (CNI) that is similar in structure to cyclosporine. It is proposed as an addition to standard induction therapy for the treatment of active lupus nephritis in adult patients.
Remestemcel-L for Acute Graft-Versus-Host Disease
Remestemcel-L is an 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.
Enspryng (Satralizumab-mwge) for Neuromyelitis Optica Spectrum Disorder
Enspryng (satralizumab) is interleukin-6 (IL-6) receptor antagonist indicated for the treatment of neuromyelitis optica spectrum disorder (NMOSD) in adult patients who are anti-aquaporin-4 (AQP4) antibody positive. It is administered by subcutaneous injection.
Viltepso (Viltolarsen) for Duchenne Muscular Dystrophy
Viltepso (viltolarsen) is an intravenously administered antisense oligonucleotide indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 53 skipping.
Evrysdi (Risdiplam) for Spinal Muscular Atrophy
Evrysdi (risdiplam; formerly RG7916) is an oral small molecule splicing modifier of the survival motor neuron 2 (SMN2) gene indicated for the treatment of spinal muscular atrophy (SMA) in patients 2 months of age and older.
Anifrolumab for Systemic Lupus Erythematosus
Anifrolumab is an investigational fully human monoclonal antibody that binds to subunit 1 of the type I interferon (IFN) receptor, blocking the activity of all type I IFNs. It is proposed for the treatment of adults with moderately to severely active systemic lupus erythematosus (SLE) who are receiving standard care.