Vyondys 53 is an antisense oligonucleotide of the phosphorodiamidate morpholino oligomer subclass intended to skip exon 53 of the DMD gene and enable the synthesis of a truncated but functional dystrophin protein. Only patients with Duchenne muscular dystrophy carrying a mutation in exon 53 will be amenable to this treatment.
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?
Inrebic (Fedratinib) for Myelofibrosis
Inrebic is an oral kinase inhibitor indicated for the treatment of adult patients with intermediate-2 or high-risk primary or secondary (post-polycythemia vera or post-essential thrombocythemia) myelofibrosis.
Inrebic (Fedratinib) for Myelofibrosis
Inrebic is an oral kinase inhibitor indicated for the treatment of adult patients with intermediate-2 or high-risk primary or secondary (post-polycythemia vera or post-essential thrombocythemia) myelofibrosis.
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.
Oxbryta (Voxelotor) for Sickle Cell Disease
Oxbryta (voxelotor) is a first-in-class oral hemoglobin oxygen affinity modulator that is believed to target and bind to the N-terminal valine of the alpha chain of hemoglobin S (HbS). This improves hemoglobin oxygen affinity and consequently inhibits HbS polymerization, thereby reducing sickling and hemolysis, and increasing the half-life of red blood cells. This is believed…
Oxbryta (Voxelotor) for Sickle Cell Disease
Oxbryta (voxelotor) is a first-in-class oral hemoglobin oxygen affinity modulator that is believed to target and bind to the N-terminal valine of the alpha chain of hemoglobin S (HbS). This improves hemoglobin oxygen affinity and consequently inhibits HbS polymerization, thereby reducing sickling and hemolysis, and increasing the half-life of red blood cells. This is believed…
Cablivi (Caplacizumab-yhdp)
Caplacizumab is a subcutaneously injected biologic agent intended for use as an adjunct to standard care for acquired thrombotic thrombocytopenic purpura. It is a bivalent single-domain anti-von Willebrand factor (VWF) nanobody that inhibits the binding of VWF to platelets to prevent the early stages of blood clot formation. Nanobodies are much smaller than conventional monoc…
Cablivi (Caplacizumab-yhdp)
Caplacizumab is a subcutaneously injected biologic agent intended for use as an adjunct to standard care for acquired thrombotic thrombocytopenic purpura. It is a bivalent single-domain anti-von Willebrand factor (VWF) nanobody that inhibits the binding of VWF to platelets to prevent the early stages of blood clot formation. Nanobodies are much smaller than conventional monoc…
Givlaari (Givosiran) for Acute Hepatic Porphyria
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…
Adakveo (Crizanlizumab-tmca) for Sickle Cell Disease
Adakveo (crizanlizumab-tmca) is an intravenous humanized monoclonal antibody that binds to P-selectin. P-selectin is an adhesion molecule expressed on vascular endothelial cells and platelets and is a key modulator in the vaso-occlusive process. The inhibition of P-selectin is believed to reduce sickle red blood cell adhesion, thereby improving blood flow and minimizing vaso-…