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).
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.
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.
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.
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.
Dexamethasone for Treatment of COVID-19
Dexamethasone is a glucocorticoid that is FDA-approved for numerous indications; it is under investigation for the treatment of COVID-19. Glucocorticoids are adrenocortical steroids that modify the body’s immune responses to diverse stimuli. Dexamethasone is indicated for treatment of allergic states, dermatologic diseases, endocrine disorders, gastrointestinal diseases, hema…
InterAtrial Shunt Device for Heart Failure
The interatrial shunt device (IASD) is a transcatheter interatrial shunt for treatment of heart failure with preserved or borderline reduced ejection fraction. It is proposed to decrease elevated left atrial pressure in patients with heart failure and preserved or borderline reduced ejection fraction. The device is implanted within the septum separating the two atrial chamber…
Ayvakit (Avapritinib) for Gastrointestinal Stromal Tumor
Ayvakit (avapritinib) is an oral potent and selective inhibitor of activated KIT and PDGFRA mutant kinases, particularly activation loop mutations and highly resistant PDGFRA D842V mutation. It is indicated for the treatment of adults with unresectable or metastatic gastrointestinal s…
SER-109 for Fecal Microbiota Transplantation
SER-109 is an investigational microbiota-based product for fecal microbiota transplantation (FMT). It is proposed to prevent recurrent Clostridioides (formerly Clostridium) difficile infection (CDI) in adults with a history of 3 or more CDI episodes within a 9-month period. SER-109 is…