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.
Horizon Scanning
It is a challenge for health plans and clinicians to know what emerging technologies are in the preapproval phase and what their clinical, financial, and risk impacts might be. This is where we come in.The Hayes Horizon Scanning Solution identifies innovative and potentially disruptive health technologies that are in various stages of the regulatory approval process. Our Research Analysts continually investigate new technologies that are on the horizon or in the early stages of clinical investigation, as well as the testing of existing technologies that may be seeking approval for new clinical applications. Hayes Horizon Scanning helps you track emerging technologies before you need to make decisions. Accessed through the Hayes Knowledge Center, Hayes Horizon Scanning addresses the following fundamental questions to help our clients forecast emerging technologies:
- Is this emerging technology new or replacing an existing technology?
- Where is this technology in the FDA regulatory process?
- When will this technology be commercially available in the market?
- What is the projected use, cost, and financial impact of this technology?
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