Berotralstat is an oral plasma kallikrein inhibitor indicated for prophylaxis to prevent attacks of hereditary angioedema (HAE) in adults and pediatric patients 12 years and older.
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.
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.
Tazverik (Tazemetostat) for Epithelioid Sarcoma
Tazverik is an oral methyltransferase inhibitor indicated for the treatment of adults and pediatric patients aged 16 years and older with metastatic or locally advanced epithelioid sarcoma not eligible for complete resection. This indication was approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication …
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.
Retevmo (Selpercatinib) for Thyroid Cancer
Retevmo (selpercatinib) is a novel oral highly selective RET inhibitor for the treatment of adult and pediatric patients 12 years of age and older with advanced or metastatic RET-mutant medullary thyroid cancer (MTC) who require systemic therapy, and adult and pediatric patients 12 years of age and older with advanced or metastatic
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). 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. 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… 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… Fenfluramine is a selective serotonin reuptake inhibitor (SSRI) indicated for the treatment of seizures associated with Dravet syndrome in patients aged 2 years and older.Retevmo (Selpercatinib) for Non-Small Cell Lung Cancer (NSCLC)
Enspryng (Satralizumab-mwge) for Neuromyelitis Optica Spectrum Disorder
Dexamethasone for Treatment of COVID-19
Ayvakit (Avapritinib) for Gastrointestinal Stromal Tumor
Fintepla (Fenfluramine) for Seizures Associated with Dravet Syndrome