Givlaari is a subcutaneously administered ribonucleic acid interference (RNAi) agent that inhibits delta-aminolevulinate synthase 1 (ALAS1) synthesis, a key regulator of the heme biosynthetic pathway in the liver. Givlaari is believed to reduce ALAS1 levels, thereby decreasing delta-aminolevulinic acid (ALA) and porphobilinogen (PBG) levels. Decreasing ALA and PBG levels to n…
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.
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. This patient…
Chloroquine and Hydroxychloroquine for COVID-19
Chloroquine (and its metabolite, hydroxychloroquine) is an oral 4-aminoquinoline antimalarial agent that has demonstrated antiviral effects on SARS-CoV and SARS-CoV-2 in vitro. It is being evaluated for the treatment of COVID-19.
Uplizna (Inebilizumab-cdon) for Neuromyelitis Optica Spectrum Disorder
Inebilizumab is an anti-CD19 monoclonal antibody. It was developed in response to the unmet treatment needs of patients with neuromyelitis optica spectrum disorder (NMOSD), a rare and debilitating autoimmune demyelinating disease that primary affects the optic nerves and spinal cord. Inebilizumab is administered every 6 months by intravenous infusion.
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.
Remdesivir for COVID-19
Remdesivir is an investigational, intravenously administered antiviral agent proposed for treatment of COVID-19.
Satralizumab for Neuromyelitis Optica Spectrum Disorder
Satralizumab is an investigational anti-IL-6 receptor monoclonal antibody under FDA review for the treatment of neuromyelitis optica spectrum disorder in adults and adolescents. It is administered by subcutaneous injection.
Glu-Plasminogen (Proposed Name: Ryplazim) for Congenital Plasminogen Deficiency
Ryplazim is an investigational intravenous plasminogen replacement therapy for treatment of congenital plasminogen deficiency in children and adults. Ryplazim is recovered from healthy donor plasma; plasminogen is extracted using a proprietary process. Purified isolated plasminogen is administered by intravenous infusion.
Chloroquine and Hydroxychloroquine for COVID-19
Chloroquine (and its metabolite, hydroxychloroquine) is an oral 4-aminoquinoline antimalarial agent that has demonstrated antiviral effects on SARS-CoV and SARS-CoV-2 in vitro. It is being evaluated for the treatment of COVID-19.
Trodelvy (sacituzumab govitecan-hziy) for Metastatic Triple Negative Breast Cancer
Trodelvy (sacituzumab govitecan-hziy) is an intravenously administered antibody-drug conjugate (ADC) indicated for the treatment of adult patients with metastatic triple-negative breast cancer (mTNBC) who have received at least 2 prior therapies for metastatic disease. Trodelvy is the first FDA-approved ADC indicated for the treatment of TNBC.