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.
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.
Remdesivir for COVID-19
Remdesivir is an investigational, intravenously administered antiviral agent proposed for treatment of COVID-19.
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.
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.
Reblozyl (Luspatercept) for Beta Thalassemia
Reblozyl is an erythroid maturation agent administered via subcutaneous injection indicated for the treatment of patients with transfusion-dependent beta thalassemia. This recombinant fusion protein is believed to help immature cells develop into red blood cells, thereby preventing anemia and reducing or eliminating the need for frequent blood transfusions.
Lopinavir/Ritonavir for COVID-19
Lopinavir/ritonavir is an oral antiretroviral protease inhibitor combination agent under investigation for the treatment of COVID-19. Lopinavir/ritonavir is FDA approved for use in combination with other antiretroviral agents for the treatment of HIV-1 infection in adults and pediatric patients aged 14 days and older.
Convalescent Plasma for Treatment of COVID-19
Convalescent plasma is a form of passive antibody therapy that is under investigation for the treatment of COVID-19. Convalescent plasma is collected from patients who have recovered from COVID-19 and administered to patients with active, symptomatic disease via intravenous infusion.